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Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment (AIM-IT)

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ClinicalTrials.gov Identifier: NCT02055118
Recruitment Status : Completed
First Posted : February 4, 2014
Results First Posted : December 13, 2018
Last Update Posted : May 16, 2019
Sponsor:
Information provided by (Responsible Party):
Shire

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hunter Syndrome
Interventions Biological: idursulfase-IT
Other: No IT treatment
Enrollment 58
Recruitment Details Study was conducted at 9 centers in Australia, Canada, France, Mexico, Spain, the United Kingdom and the United States between 24 Mar 2014 (first participant first visit) and 28 Sep 2017 (last participant last visit).
Pre-assignment Details Overall, 103 participants were screened, of them 54 participants failed to meet the randomization and remaining 49 participants were randomized to receive either IT treatment or No IT treatment in pivotal study. A total of 9 participants were enrolled into the substudy.
Arm/Group Title No IT Treatment IT Treatment Substudy
Hide Arm/Group Description Participants aged 3 to less than (<) 18 years received Elaprase therapy as standard of care for 12 months. Participants aged 3 to < 18 years received intrathecal (IT) injections of 10 milligram (mg) Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S intrathecal drug delivery device (IDDD) along with Elaprase. Participants aged less than 3 years received Idursulfase-IT monthly once for 12 months along with Elaprase at a dose of 5 mg if aged less than or equal to (<=) 8 months; 7.5 mg if aged greater than (>) 8 to 30 months and 10 mg if aged > 30 months to 3 years.
Period Title: Overall Study
Started 15 34 9
Completed 15 32 9
Not Completed 0 2 0
Reason Not Completed
Withdrawal by Subject             0             2             0
Arm/Group Title No IT Treatment IT Treatment Substudy Total
Hide Arm/Group Description Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months. Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase. Participants aged less than 3 years received Idursulfase-IT monthly once for 12 months along with Elaprase at a dose of 5 mg if aged <= 8 months; 7.5 mg if aged > 8 to 30 months and 10 mg if aged > 30 months to 3 years. Total of all reporting groups
Overall Number of Baseline Participants 15 34 9 58
Hide Baseline Analysis Population Description
Intent to treat (ITT) population included all randomized participants.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 15 participants 34 participants 9 participants 58 participants
5.29  (2.624) 4.95  (1.496) 2.48  (0.483) 4.65  (1.980)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 34 participants 9 participants 58 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
15
 100.0%
34
 100.0%
9
 100.0%
58
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 34 participants 9 participants 58 participants
Hispanic or Latino
5
  33.3%
9
  26.5%
1
  11.1%
15
  25.9%
Not Hispanic or Latino
8
  53.3%
25
  73.5%
8
  88.9%
41
  70.7%
Unknown or Not Reported
2
  13.3%
0
   0.0%
0
   0.0%
2
   3.4%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Race Number Analyzed 15 participants 34 participants 9 participants 58 participants
Asian
0
   0.0%
4
  11.8%
0
   0.0%
4
   6.9%
Black or African American
0
   0.0%
1
   2.9%
1
  11.1%
2
   3.4%
White
12
  80.0%
23
  67.6%
7
  77.8%
42
  72.4%
Other
3
  20.0%
6
  17.6%
1
  11.1%
10
  17.2%
1.Primary Outcome
Title Change From Baseline in the Differential Ability Scales, Second Edition (DAS-II) General Conceptual Ability (GCA) Standard Score at Week 52
Hide Description The DAS-II was used to assess cognitive development in all randomized participants. The GCA standard score of the DAS-II was used to obtain a general measure of cognitive ability. The GCA score represent a score (mean = 100 and standard deviation of 15) on which higher scores indicate a higher level of cognitive ability. The score ranges from 30 to 170. A positive change value indicates improvement in cognitive ability.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with number of participants evaluable for this outcome measure.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 29
Least Squares Mean (Standard Error)
Unit of Measure: Score on a scale
-7.4  (4.22) -4.4  (3.14)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection No IT Treatment, IT Treatment
Comments The Mixed model repeated measures included fixed categorical effects for treatment, visit week, treatment by visit week interaction, baseline GCA classification factor (less than or equal to [<=] 70 or >70), baseline age group (<6 years or >=6 years), treatment by baseline GCA classification factor interaction, treatment by baseline age group interaction, interaction between baseline GCA classification factor and baseline age group, genotype, and the baseline GCA score as a continuous covariate.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5669
Comments [Not Specified]
Method Mixed model repeated measures
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean
Estimated Value 3.0
Confidence Interval (2-Sided) 95%
-7.3 to 13.3
Parameter Dispersion
Type: Standard Error of the Mean
Value: 5.12
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change From Baseline in the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Adaptive Behavior Composite (ABC) Score at Week 52
Hide Description The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following 4 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite (a composite of the other 4 domains). The ABC score ranges from 20 to 160 on which higher scores indicate a higher level of adaptive functioning. A positive change value indicates improvement in adaptive functioning.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with number of participants evaluable for this outcome measure.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 14 31
Least Squares Mean (Standard Error)
Unit of Measure: Score on a scale
-5.3  (2.55) -5.0  (2.05)
3.Secondary Outcome
Title Change From Baseline in the Differential Ability Scales, Second Edition (DAS-II) General Conceptual Ability (GCA) Standard Score at Weeks 16, 28 and 40
Hide Description The DAS-II was used to assess cognitive development in all randomized participants. The GCA standard score of the DAS-II was used to obtain a general measure of cognitive ability. The GCA score represent a score (mean = 100 and standard deviation of 15) on which higher scores indicate a higher level of cognitive ability. The score ranges from 30 to 170. A positive change value indicates improvement in cognitive ability.
Time Frame Baseline, Week 16, Week 28 and Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 34
Least Squares Mean (Standard Error)
Unit of Measure: Score on a scale
Week 16 Number Analyzed 14 participants 30 participants
-2.9  (3.33) -0.6  (2.54)
Week 28 Number Analyzed 12 participants 30 participants
-6.1  (3.34) -0.3  (2.56)
Week 40 Number Analyzed 15 participants 30 participants
-6.4  (3.87) -3.2  (2.92)
4.Secondary Outcome
Title Change From Baseline in the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Adaptive Behavior Composite (ABC) Score at Week 16, 28 and 40
Hide Description The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following 4 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite (a composite of the other 4 domains). The ABC score ranges from 20 to 160 on which higher scores indicate a higher level of adaptive functioning. A positive change value indicates improvement in adaptive functioning.
Time Frame Baseline, Week 16, Week 28 and Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 34
Least Squares Mean (Standard Error)
Unit of Measure: Score on a scale
Week 16 Number Analyzed 15 participants 31 participants
-2.8  (2.21) -3.1  (1.86)
Week 28 Number Analyzed 15 participants 29 participants
-1.1  (2.45) -1.8  (2.01)
Week 40 Number Analyzed 14 participants 30 participants
-3.3  (2.41) -4.7  (1.97)
5.Secondary Outcome
Title Change From Baseline in Differential Ability Scales, Second Edition (DAS-II) Cluster Standard Scores at Weeks 16, 28, 40 and 52
Hide Description The DAS-II was used to assess cognitive development in all randomized participants. The cluster scores represent a score (mean = 100 and standard deviation of 15) on which higher scores indicate a higher level of cognitive ability in each cluster: verbal (score range: 31-169), nonverbal (score range: 31-166) and spatial (score range: 32-170).
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 34
Least Squares Mean (Standard Error)
Unit of Measure: Score on a scale
Week 16: Verbal Number Analyzed 15 participants 32 participants
-1.1  (3.75) -6.9  (2.81)
Week 16: Nonverbal Number Analyzed 14 participants 32 participants
-6.7  (5.06) 2.6  (3.82)
Week 16: Spatial Number Analyzed 11 participants 28 participants
-5.6  (3.18) 1.9  (2.47)
Week 16: Special Nonverbal Composite (SNC) Number Analyzed 11 participants 28 participants
-7.4  (3.95) -0.7  (2.96)
Week 28: Verbal Number Analyzed 12 participants 30 participants
-7.0  (4.26) -5.1  (3.10)
Week 28: Nonverbal Number Analyzed 12 participants 30 participants
-8.8  (5.41) 0.7  (4.06)
Week 28: Spatial Number Analyzed 9 participants 27 participants
-7.8  (3.66) -0.8  (2.70)
Week 28: Special Nonverbal Composite (SNC) Number Analyzed 9 participants 27 participants
-8.0  (4.01) -1.7  (3.00)
Week 40: Verbal Number Analyzed 15 participants 30 participants
-7.3  (4.01) -10.8  (3.07)
Week 40: Nonverbal Number Analyzed 15 participants 30 participants
-8.7  (5.35) -2.8  (4.09)
Week 40: Spatial Number Analyzed 11 participants 27 participants
-7.3  (4.39) 0.6  (3.13)
Week 40: Special Nonverbal Composite (SNC) Number Analyzed 11 participants 27 participants
-9.7  (5.51) -3.1  (3.78)
Week 52: Verbal Number Analyzed 15 participants 30 participants
-5.7  (4.45) -9.8  (3.31)
Week 52: Nonverbal Number Analyzed 15 participants 30 participants
-11.2  (5.61) -1.5  (4.25)
Week 52: Spatial Number Analyzed 11 participants 26 participants
-9.6  (4.21) -4.8  (3.04)
Week 52: Special Nonverbal Composite (SNC) Number Analyzed 11 participants 26 participants
-11.8  (5.38) -5.0  (3.74)
6.Secondary Outcome
Title Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Standard Scores of Other Domains at Weeks 16, 28, 40, 52
Hide Description The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following 4 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite (a composite of the other 4 domains). The standard scores represent a score (mean = 100 and standard deviation of 15) on which higher scores indicate a higher level of cognitive ability. A positive change value indicates improvement in adaptive functioning. Communication, daily living skills, socialization and motor skills domains were reported here. The range for individual standard scores is 20-160.
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 34
Least Squares Mean (Standard Error)
Unit of Measure: Score on a scale
Week 16: Communication Number Analyzed 15 participants 33 participants
-2.8  (2.65) -1.7  (1.94)
Week 16: Daily Living Skills Number Analyzed 15 participants 33 participants
-3.5  (3.11) -3.5  (2.33)
Week 16: Socialization Number Analyzed 15 participants 32 participants
-4.0  (2.43) -2.0  (2.05)
Week 16: Motor Skills Number Analyzed 12 participants 27 participants
1.0  (2.00) 0.0  (1.46)
Week 28: Communication Number Analyzed 15 participants 30 participants
-2.4  (2.80) -0.3  (2.08)
Week 28: Daily Living Skills Number Analyzed 15 participants 30 participants
0.1  (3.40) -2.0  (2.53)
Week 28: Socialization Number Analyzed 15 participants 30 participants
-0.8  (2.37) -0.2  (2.02)
Week 28: Motor Skills Number Analyzed 12 participants 25 participants
-0.2  (2.46) 0.7  (1.77)
Week 40: Communication Number Analyzed 14 participants 32 participants
-3.5  (2.82) -4.6  (2.07)
Week 40: Daily Living Skills Number Analyzed 15 participants 31 participants
-2.0  (3.47) -3.7  (2.57)
Week 40: Socialization Number Analyzed 15 participants 32 participants
-4.0  (2.65) -3.8  (2.17)
Week 40: Motor Skills Number Analyzed 12 participants 26 participants
-2.2  (2.47) -1.1  (1.77)
Week 52: Communication Number Analyzed 15 participants 32 participants
-4.8  (2.99) -5.8  (2.18)
Week 52: Daily Living Skills Number Analyzed 14 participants 32 participants
-4.5  (3.50) -4.4  (2.57)
Week 52: Socialization Number Analyzed 15 participants 32 participants
-5.8  (2.72) -2.8  (2.21)
Week 52: Motor Skills Number Analyzed 12 participants 26 participants
-5.7  (2.47) -1.3  (1.77)
7.Secondary Outcome
Title Change From Baseline of Age Equivalent Score for Early Years of Core Subtests of the Differential Ability Scales, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
Hide Description The DAS-II was used to assess cognitive development in all randomized participants. The early years battery is designed for children ages 2 years 6 months through 6 years 11 months. Standardized scores were converted to age equivalent scores to measure ability, skill, and knowledge expressed as the age at which most individuals reach the same level. The mean age equivalent score is obtained by averaging out the age-equivalent scores. The higher score indicates greater cognitive ability. The subtest score represent a score (mean = 50 and standard deviation of 10) on which higher scores indicate a higher level of cognitive ability. A positive change value indicates improvement in cognitive ability.
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with number of participants evaluable for this outcome measure.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 32
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Week 16: Verbal Comprehension Number Analyzed 14 participants 29 participants
0.05  (0.748) 0.03  (0.700)
Week 16: Picture Similarities Number Analyzed 14 participants 29 participants
0.00  (1.056) 0.48  (1.002)
Week 16: Naming Vocabulary Number Analyzed 14 participants 29 participants
0.18  (0.432) -0.02  (0.702)
Week 16: Pattern Construction Number Analyzed 14 participants 27 participants
-0.14  (0.435) 0.25  (0.475)
Week 16: Matrices Number Analyzed 10 participants 27 participants
0.48  (2.076) 0.38  (1.322)
Week 16: Copying Number Analyzed 10 participants 27 participants
0.05  (0.197) 0.10  (0.211)
Week 28: Verbal Comprehension Number Analyzed 11 participants 28 participants
-0.16  (0.846) 0.39  (0.780)
Week 28: Picture Similarities Number Analyzed 11 participants 28 participants
0.39  (1.051) 0.43  (0.693)
Week 28: Naming Vocabulary Number Analyzed 11 participants 28 participants
0.27  (0.325) 0.26  (0.870)
Week 28: Pattern Construction Number Analyzed 11 participants 28 participants
-0.18  (0.389) 0.29  (0.543)
Week 28: Matrices Number Analyzed 8 participants 25 participants
0.78  (1.089) 0.51  (1.802)
Week 28: Copying Number Analyzed 8 participants 25 participants
0.06  (0.291) 0.14  (0.361)
Week 40: Verbal Comprehension Number Analyzed 14 participants 27 participants
-0.11  (0.783) 0.17  (0.519)
Week 40: Picture Similarities Number Analyzed 14 participants 27 participants
0.23  (1.277) 0.52  (1.081)
Week 40: Naming Vocabulary Number Analyzed 14 participants 27 participants
-0.04  (0.950) 0.17  (0.721)
Week 40: Pattern Construction Number Analyzed 14 participants 27 participants
-0.02  (0.317) 0.43  (0.733)
Week 40: Matrices Number Analyzed 10 participants 24 participants
0.15  (1.890) 0.45  (1.721)
Week 40: Copying Number Analyzed 10 participants 24 participants
0.33  (0.472) 0.30  (0.556)
Week 52: Verbal Comprehension Number Analyzed 14 participants 27 participants
0.16  (1.090) 0.32  (0.657)
Week 52: Picture Similarities Number Analyzed 14 participants 27 participants
0.27  (1.207) 0.84  (1.215)
Week 52: Naming Vocabulary Number Analyzed 14 participants 27 participants
0.16  (0.959) 0.34  (1.006)
Week 52: Pattern Construction Number Analyzed 14 participants 27 participants
0.02  (0.360) 0.31  (0.660)
Week 52: Matrices Number Analyzed 10 participants 24 participants
0.00  (1.863) 0.64  (1.771)
Week 52: Copying Number Analyzed 10 participants 23 participants
0.20  (0.438) 0.28  (0.502)
8.Secondary Outcome
Title Change From Baseline of Age Equivalents for School Age of Core Subtests of the Differential Ability Scales, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
Hide Description The DAS-II was used to assess cognitive development in all randomized participants. The school age battery is designed for children ages 7 years 0 months through 17 years 11 months. Standardized scores were converted to age equivalent scores to measure ability, skill, and knowledge expressed as the age at which most individuals reach the same level. The mean age equivalent score is obtained by averaging out the age-equivalent scores. The higher score indicates greater cognitive ability. The subtest score represents a score (mean = 50 and standard deviation of 10) on which higher scores indicate a higher level of cognitive ability. A positive change value indicates improvement in cognitive ability. In the below table, SQR stands for "Sequential & Quantitative Reasoning".
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with number of participants evaluable for this outcome measure.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 1 4
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Week 16: Recall of Designs Number Analyzed 1 participants 2 participants
-1.75 [1]   (NA) 1.00  (1.414)
Week 16: Word Definitions Number Analyzed 1 participants 2 participants
1.50 [1]   (NA) 0.25  (0.707)
Week 16: Pattern Construction Number Analyzed 1 participants 2 participants
1.00 [1]   (NA) -0.13  (0.177)
Week 16: Matrices Number Analyzed 1 participants 2 participants
-0.75 [1]   (NA) 0.38  (2.652)
Week 16: Verbal Similarities Number Analyzed 1 participants 2 participants
0.75 [1]   (NA) 0.63  (0.884)
Week 16: SQR Number Analyzed 1 participants 2 participants
1.17 [1]   (NA) 0.63  (0.884)
Week 28: Recall of Designs Number Analyzed 1 participants 1 participants
-1.00 [1]   (NA) 0.00 [1]   (NA)
Week 28: Word Definitions Number Analyzed 1 participants 1 participants
1.50 [1]   (NA) -0.25 [1]   (NA)
Week 28: Pattern Construction Number Analyzed 1 participants 1 participants
1.50 [1]   (NA) -1.75 [1]   (NA)
Week 28: Matrices Number Analyzed 1 participants 1 participants
-2.50 [1]   (NA) 0.00 [1]   (NA)
Week 28: Verbal Similarities Number Analyzed 1 participants 1 participants
0.75 [1]   (NA) 0.00 [1]   (NA)
Week 28: SQR Number Analyzed 1 participants 1 participants
0.25 [1]   (NA) 0.75 [1]   (NA)
Week 40: Recall of Designs Number Analyzed 1 participants 1 participants
-2.00 [1]   (NA) 0.00 [1]   (NA)
Week 40: Word Definitions Number Analyzed 1 participants 1 participants
1.50 [1]   (NA) -0.25 [1]   (NA)
Week 40: Pattern Construction Number Analyzed 1 participants 1 participants
1.92 [1]   (NA) 0.00 [1]   (NA)
Week 40: Matrices Number Analyzed 1 participants 1 participants
-2.25 [1]   (NA) 1.25 [1]   (NA)
Week 40: Verbal Similarities Number Analyzed 1 participants 1 participants
0.75 [1]   (NA) 1.00 [1]   (NA)
Week 40: SQR Number Analyzed 1 participants 1 participants
1.17 [1]   (NA) 1.00 [1]   (NA)
Week 52: Recall of Designs Number Analyzed 1 participants 1 participants
-2.50 [1]   (NA) 0.00 [1]   (NA)
Week 52: Word Definitions Number Analyzed 1 participants 1 participants
1.75 [1]   (NA) -0.25 [1]   (NA)
Week 52: Pattern Construction Number Analyzed 1 participants 1 participants
1.92 [1]   (NA) -2.75 [1]   (NA)
Week 52: Matrices Number Analyzed 1 participants 1 participants
-0.25 [1]   (NA) 0.00 [1]   (NA)
Week 52: Verbal Similarities Number Analyzed 1 participants 1 participants
1.17 [1]   (NA) 0.00 [1]   (NA)
Week 52: SQR Number Analyzed 1 participants 1 participants
0.67 [1]   (NA) 1.00 [1]   (NA)
[1]
Standard deviation was not calculated due to less number of participants.
9.Secondary Outcome
Title Change From Baseline of Development Quotients for Early Years of Core Subtests of the Differential Ability Scales, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
Hide Description The DAS-II was used to assess cognitive development in all randomized participants. The early years battery is designed for children ages 2 years 6 months through 6 years 11 months. The DQ is a means to express a neurodevelopmental/cognitive delay. The DQ was computed as a ratio and expressed as a percentage using the age-equivalent score divided by the age at testing ([age-equivalent score/chronological age] × 100; range, 0, 100). The higher score indicates greater cognitive ability. The subtest score represent a score (mean = 50 and standard deviation of 10) on which higher scores indicate a higher level of cognitive ability. A positive change value indicates improvement in cognitive ability.
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with number of participants evaluable for this outcome measure.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 14 32
Mean (Standard Deviation)
Unit of Measure: Percentage of chronological age
Week 16: Verbal Comprehension Number Analyzed 14 participants 29 participants
-5.91  (13.454) -4.71  (12.990)
Week 16: Picture Similarities Number Analyzed 14 participants 29 participants
-4.91  (16.959) 3.41  (19.157)
Week 16: Naming Vocabulary Number Analyzed 14 participants 29 participants
-2.09  (7.758) -5.44  (11.221)
Week 16: Pattern Construction Number Analyzed 14 participants 27 participants
-7.79  (7.339) -0.43  (9.213)
Week 16: Matrices Number Analyzed 10 participants 27 participants
4.60  (30.134) 0.57  (24.054)
Week 16: Copying Number Analyzed 10 participants 27 participants
-4.59  (4.206) -4.15  (4.794)
Week 28: Verbal Comprehension Number Analyzed 11 participants 28 participants
-14.05  (16.558) -1.46  (15.790)
Week 28: Picture Similarities Number Analyzed 11 participants 28 participants
-1.89  (17.409) 0.03  (14.733)
Week 28: Naming Vocabulary Number Analyzed 11 participants 28 participants
-3.78  (6.970) -3.76  (18.235)
Week 28: Pattern Construction Number Analyzed 11 participants 28 participants
-11.95  (7.039) -3.09  (10.708)
Week 28: Matrices Number Analyzed 8 participants 25 participants
2.96  (18.576) 0.45  (34.931)
Week 28: Copying Number Analyzed 8 participants 25 participants
-6.88  (5.759) -6.66  (7.820)
Week 40: Verbal Comprehension Number Analyzed 14 participants 27 participants
-15.40  (17.544) -8.97  (11.618)
Week 40: Picture Similarities Number Analyzed 14 participants 27 participants
-6.86  (19.775) -3.11  (20.029)
Week 40: Naming Vocabulary Number Analyzed 14 participants 27 participants
-10.02  (13.307) -8.96  (14.427)
Week 40: Pattern Construction Number Analyzed 14 participants 27 participants
-11.56  (6.989) -3.89  (13.394)
Week 40: Matrices Number Analyzed 10 participants 24 participants
-7.79  (24.750) -5.73  (32.283)
Week 40: Copying Number Analyzed 10 participants 24 participants
-6.15  (9.533) -7.69  (11.208)
Week 52: Verbal Comprehension Number Analyzed 14 participants 27 participants
-14.49  (22.614) -8.59  (13.473)
Week 52: Picture Similarities Number Analyzed 14 participants 27 participants
-8.32  (19.393) 0.56  (22.325)
Week 52: Naming Vocabulary Number Analyzed 14 participants 27 participants
-10.29  (13.522) -8.20  (18.543)
Week 52: Pattern Construction Number Analyzed 14 participants 27 participants
-13.59  (8.232) -8.49  (11.528)
Week 52: Matrices Number Analyzed 10 participants 24 participants
-12.47  (23.892) -5.53  (32.584)
Week 52: Copying Number Analyzed 10 participants 23 participants
-10.19  (9.608) -10.34  (10.429)
10.Secondary Outcome
Title Change From Baseline of Development Quotients for School Age of Core Subtests of the Differential Ability Scales, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
Hide Description The DAS-II was used to assess cognitive development in all randomized participants. The school age battery is designed for children ages 7 years 0 months through 17 years 11 months. The DQ is a means to express a neurodevelopmental/cognitive delay. The DQ was computed as a ratio and expressed as a percentage using the age-equivalent score divided by the age at testing ([age-equivalent score/chronological age] × 100; range, 0, 100). The higher score indicates greater cognitive ability. The subtest score represent a score (mean = 50 and standard deviation of 10) on which higher scores indicate a higher level of cognitive ability. A positive change value indicates improvement in cognitive ability. In the below table, SQR stands for "Sequential & Quantitative Reasoning".
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with number of participants evaluable for this outcome measure.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 1 4
Mean (Standard Deviation)
Unit of Measure: Percentage of chronological age
Week 16: Recall of Designs Number Analyzed 1 participants 2 participants
-15.10 [1]   (NA) 9.20  (17.395)
Week 16: Word Definitions Number Analyzed 1 participants 2 participants
9.60 [1]   (NA) 0.00  (9.192)
Week 16: Pattern Construction Number Analyzed 1 participants 2 participants
5.70 [1]   (NA) -4.85  (2.051)
Week 16: Matrices Number Analyzed 1 participants 2 participants
-7.60 [1]   (NA) 2.40  (33.800)
Week 16: Verbal Similarities Number Analyzed 1 participants 2 participants
3.70 [1]   (NA) 4.65  (10.960)
Week 16: SQR Number Analyzed 1 participants 2 participants
6.70 [1]   (NA) 4.50  (10.748)
Week 28: Recall of Designs Number Analyzed 1 participants 1 participants
-10.40 [1]   (NA) -4.80 [1]   (NA)
Week 28: Word Definitions Number Analyzed 1 participants 1 participants
8.70 [1]   (NA) -8.20 [1]   (NA)
Week 28: Pattern Construction Number Analyzed 1 participants 1 participants
8.40 [1]   (NA) -26.90 [1]   (NA)
Week 28: Matrices Number Analyzed 1 participants 1 participants
-21.40 [1]   (NA) -3.40 [1]   (NA)
Week 28: Verbal Similarities Number Analyzed 1 participants 1 participants
2.60 [1]   (NA) -4.80 [1]   (NA)
Week 28: SQR Number Analyzed 1 participants 1 participants
-1.20 [1]   (NA) 4.40 [1]   (NA)
Week 40: Recall of Designs Number Analyzed 1 participants 1 participants
-18.70 [1]   (NA) -6.50 [1]   (NA)
Week 40: Word Definitions Number Analyzed 1 participants 1 participants
7.50 [1]   (NA) -9.90 [1]   (NA)
Week 40: Pattern Construction Number Analyzed 1 participants 1 participants
10.20 [1]   (NA) -7.20 [1]   (NA)
Week 40: Matrices Number Analyzed 1 participants 1 participants
-20.50 [1]   (NA) 10.40 [1]   (NA)
Week 40: Verbal Similarities Number Analyzed 1 participants 1 participants
1.40 [1]   (NA) 5.50 [1]   (NA)
Week 40: SQR Number Analyzed 1 participants 1 participants
4.20 [1]   (NA) 5.50 [1]   (NA)
Week 52: Recall of Designs Number Analyzed 1 participants 1 participants
-22.90 [1]   (NA) -8.10 [1]   (NA)
Week 52: Word Definitions Number Analyzed 1 participants 1 participants
8.40 [1]   (NA) -11.50 [1]   (NA)
Week 52: Pattern Construction Number Analyzed 1 participants 1 participants
9.20 [1]   (NA) -41.00 [1]   (NA)
Week 52: Matrices Number Analyzed 1 participants 1 participants
-6.90 [1]   (NA) -5.70 [1]   (NA)
Week 52: Verbal Similarities Number Analyzed 1 participants 1 participants
3.40 [1]   (NA) -8.10 [1]   (NA)
Week 52: SQR Number Analyzed 1 participants 1 participants
-0.40 [1]   (NA) 3.60 [1]   (NA)
[1]
Standard deviation was not calculated due to less number of participants.
11.Secondary Outcome
Title Change From Baseline of T-scores for Early Years of Core Subtests of the Differential Ability Scale, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
Hide Description The DAS-II was used to assess cognitive development in all randomized participants. The early years battery is designed for children ages 2 years 6 months through 6 years 11 months. The higher score indicates greater cognitive ability. The subtest score represent a score (mean = 50 and standard deviation of 10) on which higher scores indicate a higher level of cognitive ability. A positive change value indicates improvement in cognitive ability.
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with number of participants evaluable for this outcome measure.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 14 32
Mean (Standard Deviation)
Unit of Measure: T-score
Week 16: Verbal Comprehension Number Analyzed 14 participants 29 participants
-1.7  (7.27) -3.6  (9.30)
Week 16: Picture Similarities Number Analyzed 14 participants 29 participants
-2.3  (9.62) 2.1  (9.10)
Week 16: Naming Vocabulary Number Analyzed 14 participants 29 participants
-0.1  (6.01) -2.4  (6.21)
Week 16: Pattern Construction Number Analyzed 14 participants 27 participants
-6.4  (6.01) 0.6  (8.77)
Week 16: Matrices Number Analyzed 10 participants 27 participants
-4.3  (17.73) 0.4  (14.65)
Week 16: Copying Number Analyzed 10 participants 27 participants
-2.2  (8.48) -0.5  (8.76)
Week 28: Verbal Comprehension Number Analyzed 11 participants 28 participants
-2.2  (10.32) -1.8  (11.33)
Week 28: Picture Similarities Number Analyzed 11 participants 28 participants
2.6  (7.97) 0.3  (6.28)
Week 28: Naming Vocabulary Number Analyzed 11 participants 28 participants
-0.3  (4.13) -2.0  (7.79)
Week 28: Pattern Construction Number Analyzed 11 participants 28 participants
-8.9  (5.36) -1.5  (9.74)
Week 28: Matrices Number Analyzed 8 participants 25 participants
2.9  (6.47) 0.6  (16.09)
Week 28: Copying Number Analyzed 8 participants 25 participants
-0.9  (6.36) -1.6  (11.26)
Week 40: Verbal Comprehension Number Analyzed 14 participants 27 participants
-4.9  (8.21) -3.4  (6.55)
Week 40: Picture Similarities Number Analyzed 14 participants 27 participants
0.1  (13.04) -2.0  (9.81)
Week 40: Naming Vocabulary Number Analyzed 14 participants 27 participants
-4.4  (9.69) -3.9  (7.54)
Week 40: Pattern Construction Number Analyzed 14 participants 27 participants
-8.7  (5.78) -2.6  (11.24)
Week 40: Matrices Number Analyzed 10 participants 24 participants
-7.4  (16.87) -3.2  (16.71)
Week 40: Copying Number Analyzed 10 participants 24 participants
-1.8  (11.39) -0.1  (12.69)
Week 52: Verbal Comprehension Number Analyzed 14 participants 27 participants
-4.6  (11.21) -4.9  (11.10)
Week 52: Picture Similarities Number Analyzed 14 participants 27 participants
-1.7  (15.41) 0.3  (10.44)
Week 52: Naming Vocabulary Number Analyzed 14 participants 27 participants
-2.7  (8.59) -3.2  (8.54)
Week 52: Pattern Construction Number Analyzed 14 participants 27 participants
-10.9  (8.03) -6.1  (10.90)
Week 52: Matrices Number Analyzed 10 participants 24 participants
-8.5  (15.52) -0.8  (17.00)
Week 52: Copying Number Analyzed 10 participants 23 participants
-4.0  (10.68) -2.4  (12.07)
12.Secondary Outcome
Title Change From Baseline of T-scores for School Age of Core Subtests of the Differential Ability Scale, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
Hide Description The DAS-II was used to assess cognitive development in all randomized participants. The school age battery is designed for children ages 7 years 0 months through 17 years 11 months. The higher score indicates greater cognitive ability. The subtest score represent a score (mean = 50 and standard deviation of 10) on which higher scores indicate a higher level of cognitive ability. A positive change value indicates improvement in cognitive ability. In the below table, SQR stands for "Sequential & Quantitative Reasoning".
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with number of participants evaluable for this outcome measure.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 1 4
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Week 16: Recall of Designs Number Analyzed 1 participants 2 participants
-10.0 [1]   (NA) 5.0  (12.73)
Week 16: Word Definitions Number Analyzed 1 participants 2 participants
7.0 [1]   (NA) -0.5  (7.78)
Week 16: Pattern Construction Number Analyzed 1 participants 2 participants
3.0 [1]   (NA) -2.0  (1.41)
Week 16: Matrices Number Analyzed 1 participants 2 participants
-5.0 [1]   (NA) -0.5  (12.02)
Week 16: Verbal Similarities Number Analyzed 1 participants 2 participants
4.0 [1]   (NA) -4.0  (8.49)
Week 16: SQR Number Analyzed 1 participants 2 participants
2.0 [1]   (NA) 3.0  (2.83)
Week 28: Recall of Designs Number Analyzed 1 participants 1 participants
-7.0 [1]   (NA) 10.0 [1]   (NA)
Week 28: Word Definitions Number Analyzed 1 participants 1 participants
5.0 [1]   (NA) -8.0 [1]   (NA)
Week 28: Pattern Construction Number Analyzed 1 participants 1 participants
4.0 [1]   (NA) -19.0 [1]   (NA)
Week 28: Matrices Number Analyzed 1 participants 1 participants
-12.0 [1]   (NA) -9.0 [1]   (NA)
Week 28: Verbal Similarities Number Analyzed 1 participants 1 participants
3.0 [1]   (NA) -10.0 [1]   (NA)
Week 28: SQR Number Analyzed 1 participants 1 participants
-2.0 [1]   (NA) 9.0 [1]   (NA)
Week 40: Recall of Designs Number Analyzed 1 participants 1 participants
-14.0 [1]   (NA) -4.0 [1]   (NA)
Week 40: Word Definitions Number Analyzed 1 participants 1 participants
5.0 [1]   (NA) -16.0 [1]   (NA)
Week 40: Pattern Construction Number Analyzed 1 participants 1 participants
5.0 [1]   (NA) -3.0 [1]   (NA)
Week 40: Matrices Number Analyzed 1 participants 1 participants
-11.0 [1]   (NA) 1.0 [1]   (NA)
Week 40: Verbal Similarities Number Analyzed 1 participants 1 participants
2.0 [1]   (NA) 1.0 [1]   (NA)
Week 40: SQR Number Analyzed 1 participants 1 participants
1.0 [1]   (NA) 10.0 [1]   (NA)
Week 52: Recall of Designs Number Analyzed 1 participants 1 participants
-17.0 [1]   (NA) -4.0 [1]   (NA)
Week 52: Word Definitions Number Analyzed 1 participants 1 participants
6.0 [1]   (NA) -17.0 [1]   (NA)
Week 52: Pattern Construction Number Analyzed 1 participants 1 participants
4.0 [1]   (NA) -30.0 [1]   (NA)
Week 52: Matrices Number Analyzed 1 participants 1 participants
-4.0 [1]   (NA) -5.0 [1]   (NA)
Week 52: Verbal Similarities Number Analyzed 1 participants 1 participants
5.0 [1]   (NA) -10.0 [1]   (NA)
Week 52: SQR Number Analyzed 1 participants 4 participants
-2.0 [1]   (NA) 9.0 [1]   (NA)
[1]
Standard deviation was not calculated due to less number of participants.
13.Secondary Outcome
Title Change From Baseline of Age Equivalents Scores of Sub-domains of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) at Weeks 16, 28, 40 and 52
Hide Description The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. Standardized scores (range 40-160) were converted to age equivalent scores to measure ability, skill, and knowledge expressed as the age at which most individuals reach the same level. The mean age equivalent score is obtained by averaging out the age-equivalent scores for the all the sub-domains except for Gross and Fine motor skills (range: 0, unbound). A positive value indicates improvement in health and cognition.
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 34
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Week 16: Communication- Expressive Number Analyzed 15 participants 33 participants
0.09  (0.512) 0.12  (0.544)
Week 16: Communication- Receptive Number Analyzed 15 participants 33 participants
-0.09  (1.020) -0.32  (1.845)
Week 16: Communication- Written Number Analyzed 15 participants 33 participants
0.08  (0.406) 0.02  (0.572)
Week 16: Daily Living Skills- Community Number Analyzed 15 participants 33 participants
0.16  (0.583) 0.05  (0.710)
Week 16: Daily Living Skills- Domestic Number Analyzed 15 participants 33 participants
0.02  (1.477) -0.09  (1.149)
Week 16: Daily Living Skills- Personal Number Analyzed 15 participants 33 participants
-0.23  (0.864) -0.11  (0.599)
Week 16: Socialization- Coping Skills Number Analyzed 15 participants 32 participants
-0.16  (0.712) -0.09  (0.792)
Week 16: Socialization- Interpersonal Relationship Number Analyzed 15 participants 33 participants
-0.09  (0.749) 0.04  (0.637)
Week 16: Socialization- Play and Leisure Time Number Analyzed 15 participants 33 participants
0.21  (0.934) 0.11  (0.783)
Week 16: Motor Skills- Fine Number Analyzed 12 participants 27 participants
0.30  (0.499) 0.29  (0.429)
Week 16: Motor Skills- Gross Number Analyzed 12 participants 28 participants
0.17  (0.607) 0.36  (0.736)
Week 28: Communication- Expressive Number Analyzed 15 participants 30 participants
0.28  (0.669) 0.36  (0.806)
Week 28: Communication- Receptive Number Analyzed 15 participants 30 participants
0.83  (2.393) 0.44  (1.333)
Week 28: Communication- Written Number Analyzed 15 participants 30 participants
0.21  (0.663) 0.18  (0.525)
Week 28: Daily Living Skills- Community Number Analyzed 15 participants 30 participants
0.22  (0.701) 0.26  (0.762)
Week 28: Daily Living Skills- Domestic Number Analyzed 15 participants 30 participants
0.67  (1.481) 0.34  (1.326)
Week 28: Daily Living Skills- Personal Number Analyzed 15 participants 30 participants
0.48  (1.260) 0.16  (0.544)
Week 28: Socialization- Coping Skills Number Analyzed 15 participants 30 participants
0.29  (0.731) 0.27  (0.982)
Week 28: Socialization- Interpersonal Relationship Number Analyzed 15 participants 30 participants
0.07  (0.884) 0.43  (0.876)
Week 28: Socialization- Play and Leisure Time Number Analyzed 15 participants 30 participants
0.62  (0.818) 0.51  (1.017)
Week 28: Motor Skills- Fine Number Analyzed 12 participants 25 participants
0.30  (0.403) 0.37  (0.533)
Week 28: Motor Skills- Gross Number Analyzed 12 participants 26 participants
0.41  (0.588) 0.82  (1.061)
Week 40: Communication- Expressive Number Analyzed 15 participants 32 participants
0.28  (0.679) 0.30  (0.764)
Week 40: Communication- Receptive Number Analyzed 15 participants 32 participants
0.80  (2.388) -0.07  (2.015)
Week 40: Communication- Written Number Analyzed 14 participants 32 participants
0.30  (0.699) 0.00  (0.740)
Week 40: Daily Living Skills- Community Number Analyzed 15 participants 31 participants
0.38  (0.640) 0.02  (0.760)
Week 40: Daily Living Skills- Domestic Number Analyzed 15 participants 32 participants
0.72  (1.542) 0.39  (1.420)
Week 40: Daily Living Skills- Personal Number Analyzed 15 participants 32 participants
0.18  (0.720) 0.29  (0.587)
Week 40: Socialization- Coping Skills Number Analyzed 15 participants 32 participants
0.14  (1.178) 0.26  (1.581)
Week 40: Socialization- Interpersonal Relationship Number Analyzed 15 participants 32 participants
0.05  (0.796) 0.10  (0.973)
Week 40: Socialization- Play and Leisure Time Number Analyzed 15 participants 32 participants
0.44  (0.808) 0.31  (0.921)
Week 40: Motor Skills- Fine Number Analyzed 12 participants 26 participants
0.44  (0.453) 0.44  (0.640)
Week 40: Motor Skills- Gross Number Analyzed 12 participants 27 participants
0.33  (0.662) 0.73  (0.990)
Week 52: Communication- Expressive Number Analyzed 15 participants 32 participants
0.32  (0.973) 0.34  (0.858)
Week 52: Communication- Receptive Number Analyzed 15 participants 32 participants
1.25  (2.253) -0.05  (1.953)
Week 52: Communication- Written Number Analyzed 15 participants 32 participants
0.16  (0.485) 0.11  (0.783)
Week 52: Daily Living Skills- Community Number Analyzed 14 participants 32 participants
0.36  (0.603) 0.30  (0.941)
Week 52: Daily Living Skills- Domestic Number Analyzed 15 participants 32 participants
0.27  (1.678) 0.37  (1.405)
Week 52: Daily Living Skills- Personal Number Analyzed 15 participants 32 participants
0.42  (0.572) 0.35  (0.840)
Week 52: Socialization- Coping Skills Number Analyzed 15 participants 32 participants
0.29  (1.211) 0.31  (1.189)
Week 52: Socialization- Interpersonal Relationship Number Analyzed 15 participants 32 participants
0.00  (0.752) 0.36  (1.005)
Week 52: Socialization- Play and Leisure Time Number Analyzed 15 participants 32 participants
0.67  (1.446) 0.45  (0.839)
Week 52: Motor Skills- Fine Number Analyzed 12 participants 26 participants
0.38  (0.365) 0.58  (0.645)
Week 52: Motor Skills- Gross Number Analyzed 12 participants 27 participants
0.25  (0.773) 0.93  (1.008)
14.Secondary Outcome
Title Change From Baseline of Development Quotients of Sub-domains of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) at Weeks 16, 28, 40 and 52
Hide Description The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. The DQ is a means to express a neurodevelopmental/cognitive delay. The DQ was computed as a ratio and expressed as a percentage using the age-equivalent score divided by the age at testing ([age-equivalent score/chronological age] × 100; range, 0, 100). The overall DQ score is calculated from the mean age-equivalent score obtained by averaging out the age equivalent scores for the all the sub-domains except for Gross and Fine motor skills. This test measures the following 4 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite (a composite of the other 4 domains). A positive value indicates improvement in health and cognition.
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 34
Mean (Standard Deviation)
Unit of Measure: Percentage of chronological age
Week 16: Communication- Expressive Number Analyzed 15 participants 33 participants
-1.09  (7.866) -2.57  (11.781)
Week 16: Communication- Receptive Number Analyzed 15 participants 33 participants
-2.58  (14.735) -8.19  (26.774)
Week 16: Communication- Written Number Analyzed 15 participants 33 participants
-4.10  (8.727) -5.62  (12.554)
Week 16: Daily Living Skills- Community Number Analyzed 15 participants 33 participants
-0.37  (12.814) -4.48  (15.498)
Week 16: Daily Living Skills- Domestic Number Analyzed 15 participants 33 participants
-2.80  (30.338) -7.18  (24.610)
Week 16: Daily Living Skills- Personal Number Analyzed 15 participants 33 participants
-9.94  (21.052) -6.35  (11.185)
Week 16: Socialization- Coping Skills Number Analyzed 15 participants 32 participants
-9.08  (14.414) -6.56  (15.923)
Week 16: Socialization- Interpersonal Relationship Number Analyzed 15 participants 33 participants
-3.87  (9.602) -3.17  (14.610)
Week 16: Socialization- Play and Leisure Time Number Analyzed 15 participants 33 participants
1.66  (18.903) -0.64  (15.366)
Week 16: Motor Skills- Fine Number Analyzed 12 participants 27 participants
0.48  (12.969) 0.00  (10.308)
Week 16: Motor Skills- Gross Number Analyzed 12 participants 28 participants
-3.92  (15.524) 1.28  (15.410)
Week 28: Communication- Expressive Number Analyzed 15 participants 30 participants
-0.23  (11.301) -0.59  (14.480)
Week 28: Communication- Receptive Number Analyzed 15 participants 30 participants
3.30  (24.423) 0.50  (17.058)
Week 28: Communication- Written Number Analyzed 15 participants 30 participants
-5.15  (13.510) -6.54  (11.339)
Week 28: Daily Living Skills- Community Number Analyzed 15 participants 30 participants
-3.07  (13.984) -2.80  (15.937)
Week 28: Daily Living Skills- Domestic Number Analyzed 15 participants 30 participants
6.75  (28.703) -3.37  (25.635)
Week 28: Daily Living Skills- Personal Number Analyzed 15 participants 30 participants
3.41  (26.699) -4.27  (12.124)
Week 28: Socialization- Coping Skills Number Analyzed 15 participants 30 participants
-4.88  (15.881) -2.45  (19.142)
Week 28: Socialization- Interpersonal Relationship Number Analyzed 15 participants 30 participants
-2.30  (13.889) 2.16  (17.844)
Week 28: Socialization- Play and Leisure Time Number Analyzed 15 participants 30 participants
8.17  (17.767) 4.49  (20.413)
Week 28: Motor Skills- Fine Number Analyzed 12 participants 25 participants
-2.86  (9.014) -2.05  (12.124)
Week 28: Motor Skills- Gross Number Analyzed 12 participants 26 participants
-3.05  (15.068) 7.78  (21.604)
Week 40: Communication- Expressive Number Analyzed 15 participants 32 participants
-2.35  (10.521) -3.74  (12.846)
Week 40: Communication- Receptive Number Analyzed 15 participants 32 participants
0.31  (22.511) -8.32  (29.716)
Week 40: Communication- Written Number Analyzed 14 participants 32 participants
-5.39  (14.066) -13.31  (15.173)
Week 40: Daily Living Skills- Community Number Analyzed 15 participants 31 participants
-2.29  (13.856) -9.72  (15.988)
Week 40: Daily Living Skills- Domestic Number Analyzed 15 participants 32 participants
4.40  (27.464) -4.53  (27.189)
Week 40: Daily Living Skills- Personal Number Analyzed 15 participants 32 participants
-5.54  (14.516) -4.33  (11.630)
Week 40: Socialization- Coping Skills Number Analyzed 15 participants 32 participants
-11.75  (18.650) -7.31  (22.758)
Week 40: Socialization- Interpersonal Relationship Number Analyzed 15 participants 32 participants
-5.47  (13.597) -6.38  (18.307)
Week 40: Socialization- Play and Leisure Time Number Analyzed 15 participants 32 participants
1.83  (18.194) -1.20  (19.009)
Week 40: Motor Skills- Fine Number Analyzed 12 participants 26 participants
-3.51  (10.891) -4.04  (14.407)
Week 40: Motor Skills- Gross Number Analyzed 12 participants 27 participants
-9.01  (18.404) 1.75  (18.235)
Week 52: Communication- Expressive Number Analyzed 15 participants 32 participants
-4.87  (13.094) -5.22  (14.616)
Week 52: Communication- Receptive Number Analyzed 15 participants 32 participants
5.58  (18.225) -10.14  (29.190)
Week 52: Communication- Written Number Analyzed 15 participants 32 participants
-11.22  (10.973) -13.90  (16.079)
Week 52: Daily Living Skills- Community Number Analyzed 14 participants 32 participants
-6.46  (13.154) -7.10  (18.149)
Week 52: Daily Living Skills- Domestic Number Analyzed 15 participants 32 participants
-6.81  (28.704) -8.64  (27.817)
Week 52: Daily Living Skills- Personal Number Analyzed 15 participants 32 participants
-3.64  (11.626) -5.79  (15.032)
Week 52: Socialization- Coping Skills Number Analyzed 15 participants 32 participants
-11.26  (19.505) -6.54  (20.142)
Week 52: Socialization- Interpersonal Relationship Number Analyzed 15 participants 32 participants
-8.53  (12.960) -4.46  (17.892)
Week 52: Socialization- Play and Leisure Time Number Analyzed 15 participants 32 participants
1.95  (25.499) -0.83  (15.499)
Week 52: Motor Skills- Fine Number Analyzed 12 participants 26 participants
-8.58  (8.790) -4.67  (15.370)
Week 52: Motor Skills- Gross Number Analyzed 12 participants 27 participants
-14.54  (21.617) 2.46  (18.655)
15.Secondary Outcome
Title Change From Baseline of V-Scale Scores of Sub-domains of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) at Weeks 16, 28, 40 and 52
Hide Description The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following 4 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite (a composite of the other 4 domains). The V-scale scores represent a score (mean = 15 and standard deviation of 3; range: 1-24) on which higher scores indicate a higher level of adaptive functioning. A positive change value indicates improvement in adaptive functioning.
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 34
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Week 16: Communication- Expressive Number Analyzed 15 participants 33 participants
-0.1  (1.81) -0.4  (2.05)
Week 16: Communication- Receptive Number Analyzed 15 participants 33 participants
-0.3  (1.84) -0.7  (1.85)
Week 16: Communication- Written Number Analyzed 15 participants 33 participants
-0.7  (1.83) -1.1  (2.87)
Week 16: Daily Living Skills- Community Number Analyzed 15 participants 33 participants
-0.3  (1.28) -0.7  (2.05)
Week 16: Daily Living Skills- Domestic Number Analyzed 15 participants 33 participants
-0.3  (2.61) -0.7  (1.93)
Week 16: Daily Living Skills- Personal Number Analyzed 15 participants 33 participants
-1.2  (3.23) -1.2  (2.34)
Week 16: Socialization- Coping Skills Number Analyzed 15 participants 32 participants
-0.6  (1.80) -0.4  (1.46)
Week 16: Socialization- Interpersonal Relationship Number Analyzed 15 participants 33 participants
-0.5  (1.46) -0.3  (1.55)
Week 16: Socialization- Play and Leisure Time Number Analyzed 15 participants 33 participants
0.2  (2.37) -0.4  (1.62)
Week 16: Motor Skills- Fine Number Analyzed 12 participants 27 participants
0.3  (1.36) -0.1  (1.33)
Week 16: Motor Skills- Gross Number Analyzed 12 participants 28 participants
-0.3  (1.61) 0.0  (2.00)
Week 28: Communication- Expressive Number Analyzed 15 participants 30 participants
0.0  (2.00) -0.3  (2.20)
Week 28: Communication- Receptive Number Analyzed 15 participants 30 participants
-0.1  (2.40) -0.1  (1.17)
Week 28: Communication- Written Number Analyzed 15 participants 30 participants
-0.6  (2.41) -1.2  (2.04)
Week 28: Daily Living Skills- Community Number Analyzed 15 participants 30 participants
-0.7  (1.75) -0.7  (1.95)
Week 28: Daily Living Skills- Domestic Number Analyzed 15 participants 30 participants
0.4  (2.75) -0.4  (2.11)
Week 28: Daily Living Skills- Personal Number Analyzed 15 participants 30 participants
0.6  (3.50) -0.6  (2.21)
Week 28: Socialization- Coping Skills Number Analyzed 15 participants 30 participants
0.1  (1.51) -0.2  (1.43)
Week 28: Socialization- Interpersonal Relationship Number Analyzed 15 participants 30 participants
-0.4  (1.84) 0.1  (1.49)
Week 28: Socialization- Play and Leisure Time Number Analyzed 15 participants 30 participants
1.1  (2.03) 0.2  (2.04)
Week 28: Motor Skills- Fine Number Analyzed 12 participants 25 participants
-0.3  (1.06) -0.3  (1.62)
Week 28: Motor Skills- Gross Number Analyzed 12 participants 26 participants
-0.2  (2.33) 0.5  (2.06)
Week 40: Communication- Expressive Number Analyzed 15 participants 32 participants
0.0  (1.85) -0.8  (1.76)
Week 40: Communication- Receptive Number Analyzed 15 participants 32 participants
-0.2  (2.08) -0.7  (2.07)
Week 40: Communication- Written Number Analyzed 14 participants 32 participants
-0.9  (2.59) -2.6  (3.05)
Week 40: Daily Living Skills- Community Number Analyzed 15 participants 31 participants
-0.6  (1.59) -1.6  (1.91)
Week 40: Daily Living Skills- Domestic Number Analyzed 15 participants 32 participants
0.3  (2.72) -0.7  (2.66)
Week 40: Daily Living Skills- Personal Number Analyzed 15 participants 32 participants
-0.6  (2.69) -0.5  (2.09)
Week 40: Socialization- Coping Skills Number Analyzed 15 participants 32 participants
-0.6  (1.96) -0.8  (1.80)
Week 40: Socialization- Interpersonal Relationship Number Analyzed 15 participants 32 participants
-0.8  (1.70) -0.9  (1.72)
Week 40: Socialization- Play and Leisure Time Number Analyzed 15 participants 32 participants
0.5  (2.10) -0.4  (2.24)
Week 40: Motor Skills- Fine Number Analyzed 12 participants 26 participants
-0.3  (1.56) -0.5  (1.79)
Week 40: Motor Skills- Gross Number Analyzed 12 participants 27 participants
-0.8  (1.90) -0.1  (2.23)
Week 52: Communication- Expressive Number Analyzed 15 participants 32 participants
-0.5  (1.88) -1.0  (2.44)
Week 52: Communication- Receptive Number Analyzed 15 participants 32 participants
0.1  (2.00) -0.9  (2.08)
Week 52: Communication- Written Number Analyzed 15 participants 32 participants
-2.1  (2.09) -3.0  (3.25)
Week 52: Daily Living Skills- Community Number Analyzed 14 participants 32 participants
-1.5  (1.70) -1.6  (2.46)
Week 52: Daily Living Skills- Domestic Number Analyzed 15 participants 32 participants
-0.5  (2.77) -0.7  (2.40)
Week 52: Daily Living Skills- Personal Number Analyzed 15 participants 32 participants
-0.1  (1.85) -0.9  (2.76)
Week 52: Socialization- Coping Skills Number Analyzed 15 participants 32 participants
-0.7  (1.80) -0.5  (1.55)
Week 52: Socialization- Interpersonal Relationship Number Analyzed 15 participants 32 participants
-1.0  (1.65) -0.6  (1.81)
Week 52: Socialization- Play and Leisure Time Number Analyzed 15 participants 32 participants
-0.1  (3.38) -0.4  (2.00)
Week 52: Motor Skills- Fine Number Analyzed 12 participants 26 participants
-1.1  (1.08) -0.6  (2.06)
Week 52: Motor Skills- Gross Number Analyzed 12 participants 27 participants
-1.5  (2.07) -0.1  (2.35)
16.Secondary Outcome
Title Change From Baseline of V-Scale Scores of Maladaptive Behavior Index and Its Sub-scales at Weeks 16, 28, 40 and 52
Hide Description The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. Maladaptive behavior index is a composite of the internalizing, externalizing, and other types of undesirable behavior that may interfere with the individual’s adaptive functioning. The V-scale scores represent a score (mean = 15 and standard deviation of 3; range: 1-24) on which higher scores indicate greater maladaptive behaviors. A positive change value indicates increase of maladaptive behavior.
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 34
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Week 16: Maladaptive Behavior Index Number Analyzed 12 participants 31 participants
0.3  (1.14) -0.2  (1.18)
Week 16: Maladaptive Behavior Index- Internalizing Number Analyzed 14 participants 32 participants
0.1  (2.37) -0.3  (2.31)
Week 16: Maladaptive Behavior Index- Externalizing Number Analyzed 13 participants 31 participants
0.5  (1.71) 0.0  (1.53)
Week 28: Maladaptive Behavior Index Number Analyzed 14 participants 28 participants
0.3  (0.91) -0.3  (1.27)
Week 28: Maladaptive Behavior Index- Internalizing Number Analyzed 14 participants 29 participants
-0.4  (1.60) -0.8  (2.11)
Week 28: Maladaptive Behavior Index- Externalizing Number Analyzed 14 participants 28 participants
0.6  (1.34) -0.1  (2.01)
Week 40: Maladaptive Behavior Index Number Analyzed 14 participants 31 participants
-0.5  (1.40) -0.3  (1.13)
Week 40: Maladaptive Behavior Index- Internalizing Number Analyzed 14 participants 31 participants
-1.0  (2.57) -0.7  (2.98)
Week 40: Maladaptive Behavior Index- Externalizing Number Analyzed 14 participants 31 participants
-0.1  (1.64) -0.1  (1.29)
Week 52: Maladaptive Behavior Index Number Analyzed 14 participants 31 participants
-0.5  (1.45) -0.5  (1.41)
Week 52: Maladaptive Behavior Index- Internalizing Number Analyzed 14 participants 31 participants
-1.1  (1.96) -0.9  (2.72)
Week 52: Maladaptive Behavior Index- Externalizing Number Analyzed 14 participants 31 participants
-0.4  (2.27) -0.2  (1.69)
17.Secondary Outcome
Title Observed Maladaptive Levels of Maladaptive Behavior Index and Its Sub-scales of Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
Hide Description The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. Maladaptive behavior index (MBI) is a composite of the internalizing, externalizing, and other types of undesirable behavior that may interfere with the individual’s adaptive functioning. The V scale scores represent a score (mean = 15 and standard deviation of 3; range: 1-24) on which higher scores indicate greater maladaptive behaviors. The v-scale score ranges for MBI, externalizing and internalizing scores are defined as clinically significant: 21-24, elevated: 18-20, average: 1-17.
Time Frame Baseline, Week 16, Week 28, Week 40 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 34
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline: MBI- Average
7
  46.7%
8
  23.5%
Baseline: MBI- Elevated
5
  33.3%
20
  58.8%
Baseline: MBI- Clinically Significant
2
  13.3%
5
  14.7%
Baseline: Internalizing- Average
7
  46.7%
15
  44.1%
Baseline: Internalizing- Elevated
4
  26.7%
11
  32.4%
Baseline: Internalizing- Clinically Significant
3
  20.0%
7
  20.6%
Baseline: Externalizing- Average
8
  53.3%
11
  32.4%
Baseline: Externalizing- Elevated
5
  33.3%
19
  55.9%
Baseline: Externalizing- Clinically Significant
1
   6.7%
3
   8.8%
Week 16: MBI- Average
4
  26.7%
9
  26.5%
Week 16: MBI- Elevated
8
  53.3%
17
  50.0%
Week 16: MBI- Clinically Significant
1
   6.7%
6
  17.6%
Week 16: Internalizing- Average
7
  46.7%
19
  55.9%
Week 16: Internalizing- Elevated
6
  40.0%
9
  26.5%
Week 16: Internalizing- Clinically Significant
2
  13.3%
5
  14.7%
Week 16: Externalizing- Average
7
  46.7%
13
  38.2%
Week 16: Externalizing- Elevated
7
  46.7%
14
  41.2%
Week 16: Externalizing- Clinically Significant
0
   0.0%
5
  14.7%
Week 28: MBI- Average
7
  46.7%
8
  23.5%
Week 28: MBI- Elevated
7
  46.7%
16
  47.1%
Week 28: MBI- Clinically Significant
1
   6.7%
5
  14.7%
Week 28: Internalizing- Average
9
  60.0%
18
  52.9%
Week 28: Internalizing- Elevated
6
  40.0%
10
  29.4%
Week 28: Internalizing- Clinically Significant
0
   0.0%
2
   5.9%
Week 28: Externalizing- Average
10
  66.7%
11
  32.4%
Week 28: Externalizing- Elevated
5
  33.3%
14
  41.2%
Week 28: Externalizing- Clinically Significant
0
   0.0%
4
  11.8%
Week 40: MBI- Average
7
  46.7%
7
  20.6%
Week 40: MBI- Elevated
8
  53.3%
21
  61.8%
Week 40: MBI- Clinically Significant
0
   0.0%
4
  11.8%
Week 40: Internalizing- Average
9
  60.0%
20
  58.8%
Week 40: Internalizing- Elevated
6
  40.0%
10
  29.4%
Week 40: Internalizing- Clinically Significant
0
   0.0%
2
   5.9%
Week 40: Externalizing- Average
9
  60.0%
12
  35.3%
Week 40: Externalizing- Elevated
6
  40.0%
18
  52.9%
Week 40: Externalizing- Clinically Significant
0
   0.0%
2
   5.9%
Week 52: MBI- Average
9
  60.0%
10
  29.4%
Week 52: MBI- Elevated
6
  40.0%
18
  52.9%
Week 52: MBI- Clinically Significant
0
   0.0%
4
  11.8%
Week 52: Internalizing- Average
11
  73.3%
20
  58.8%
Week 52: Internalizing- Elevated
4
  26.7%
9
  26.5%
Week 52: Internalizing- Clinically Significant
0
   0.0%
3
   8.8%
Week 52: Externalizing- Average
10
  66.7%
15
  44.1%
Week 52: Externalizing- Elevated
5
  33.3%
15
  44.1%
Week 52: Externalizing- Clinically Significant
0
   0.0%
2
   5.9%
18.Secondary Outcome
Title Maximum Observed Drug Concentration (Cmax) of Idursulfase After IT Administration
Hide Description The Cmax of idursulfase after IT administration was reported.
Time Frame Pre-dose, 30, 60, 120 minutes, 4, 6, 8, 12, 24, 30 and 36 hour (h) post-dose on Weeks 4, 24, and 48
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic (PK) population included all participants who received investigational product and participated in the scheduled PK studies, and for whom at least 1 post-dose PK blood sample was collected. PK population with number of participants evaluable for this outcome measure.
Arm/Group Title IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 24
Mean (Standard Deviation)
Unit of Measure: Nanogram per milliliter (ng/mL)
Week 4 Number Analyzed 24 participants
253.44  (686.569)
Week 24 Number Analyzed 22 participants
132.87  (147.044)
Week 48 Number Analyzed 22 participants
136.15  (127.524)
19.Secondary Outcome
Title Time to Reach Maximum Drug Concentration (Tmax) of Idursulfase After IT Administration
Hide Description The tmax of idursulfase after IT administration was reported.
Time Frame Pre-dose, 30, 60, 120 minutes, 4, 6, 8, 12, 24, 30 and 36 hour (h) post-dose on Weeks 4, 24, and 48
Hide Outcome Measure Data
Hide Analysis Population Description
PK population with number of participants evaluable for this outcome measure.
Arm/Group Title IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 24
Mean (Standard Deviation)
Unit of Measure: Hour (h)
Week 4 Number Analyzed 24 participants
10.07  (7.217)
Week 24 Number Analyzed 22 participants
10.37  (7.302)
Week 48 Number Analyzed 22 participants
10.18  (6.609)
20.Secondary Outcome
Title Area Under the Concentration Versus Time Curve From Zero From the Time of Dosing to the Last Measurable Concentration (AUC0-t) of Idursulfase After IT Administration
Hide Description The AUC0-t of idursulfase after IT administration was reported.
Time Frame Pre-dose, 30, 60, 120 minutes, 4, 6, 8, 12, 24, 30 and 36 hour (h) post-dose on Weeks 4, 24, and 48
Hide Outcome Measure Data
Hide Analysis Population Description
PK population with number of participants evaluable for this outcome measure.
Arm/Group Title IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 24
Mean (Standard Deviation)
Unit of Measure: Nanogram*hour per milliliter (ng*h/mL)
Week 4 Number Analyzed 24 participants
2601  (5028.0)
Week 24 Number Analyzed 22 participants
2949  (4500.4)
Week 48 Number Analyzed 22 participants
2863  (3571.6)
21.Secondary Outcome
Title Terminal Half-life (t1/2) of Idursulfase After IT Administration
Hide Description The t1/2 of idursulfase after IT administration was reported.
Time Frame Pre-dose, 30, 60, 120 minutes, 4, 6, 8, 12, 24, 30 and 36 hour (h) post-dose on Weeks 4, 24, and 48
Hide Outcome Measure Data
Hide Analysis Population Description
PK population with number of participants evaluable for this outcome measure.
Arm/Group Title IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 24
Mean (Standard Deviation)
Unit of Measure: Hour (h)
Week 4 Number Analyzed 11 participants
11.39  (4.996)
Week 24 Number Analyzed 12 participants
10.68  (2.851)
Week 48 Number Analyzed 11 participants
11.52  (4.243)
22.Secondary Outcome
Title Total Body Clearance for Extravascular Administration Divided by the Fraction of Dose Absorbed (CL/F) of Idursulfase After IT Administration
Hide Description The CL/F of idursulfase after IT administration was reported.
Time Frame Pre-dose, 30, 60, 120 minutes, 4, 6, 8, 12, 24, 30 and 36 hour (h) post-dose on Weeks 4, 24, and 48
Hide Outcome Measure Data
Hide Analysis Population Description
PK population with number of participants evaluable for this outcome measure.
Arm/Group Title IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 24
Mean (Standard Deviation)
Unit of Measure: Liter per hour (L/h)
Week 4 Number Analyzed 11 participants
4.82  (1.753)
Week 24 Number Analyzed 12 participants
4.54  (2.029)
Week 48 Number Analyzed 11 participants
3.94  (1.461)
23.Secondary Outcome
Title Change From Baseline in the Concentration of Glycosaminoglycans (GAG) in Cerebrospinal Fluid (CSF) at Week 52
Hide Description Change from baseline in the concentration of GAG in CSF was reported.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
PK population with number of participants evaluable for this outcome measure.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 14 30
Mean (Standard Deviation)
Unit of Measure: Nanogram per milliliter (ng/mL)
-124.6  (905.08) -961.8  (797.01)
24.Secondary Outcome
Title Concentration of Idursulfase in Cerebrospinal Fluid (CSF)
Hide Description CSF samples were collected via the IDDD or lumbar puncture prior to the injection of Idursulfase-IT.
Time Frame Pre-dose on Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
PK population with number of participants evaluable for this outcome measure.
Arm/Group Title IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 33
Mean (Standard Deviation)
Unit of Measure: ng/mL
Week 4 Number Analyzed 31 participants
0  (0)
Week 8 Number Analyzed 29 participants
552.75  (2638.000)
Week 12 Number Analyzed 27 participants
520.29  (2096.512)
Week 16 Number Analyzed 27 participants
176.87  (572.459)
Week 20 Number Analyzed 29 participants
146.66  (553.354)
Week 24 Number Analyzed 29 participants
268.72  (979.733)
Week 28 Number Analyzed 28 participants
1471.61  (5893.839)
Week 32 Number Analyzed 28 participants
141.16  (443.484)
Week 36 Number Analyzed 29 participants
521.63  (1524.740)
Week 40 Number Analyzed 29 participants
14436.70  (75712.900)
Week 44 Number Analyzed 29 participants
1105.46  (5331.560)
Week 48 Number Analyzed 27 participants
678.50  (3266.592)
25.Secondary Outcome
Title Participant Response to Quality of Life EuroQol-5D (EQ-5D) Questionnaire at Week 52
Hide Description The EQ-5D provides a descriptive profile and index value for health status. The questionnaire measures 5 dimensions of health status: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. For each dimension, there are 5 levels of response: no problems, slight problems, moderate problems, severe problems, and unable to do/extreme problems.
Time Frame Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all randomized participants.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 34
Measure Type: Count of Participants
Unit of Measure: Participants
Mobility: No problems
9
  60.0%
22
  64.7%
Mobility: Slight problems
4
  26.7%
7
  20.6%
Mobility: Moderate problems
1
   6.7%
1
   2.9%
Mobility: Severe problems
0
   0.0%
0
   0.0%
Mobility: Unable to do
0
   0.0%
0
   0.0%
Mobility: Data missing
1
   6.7%
4
  11.8%
Self-care: No problems
3
  20.0%
6
  17.6%
Self-care: Slight problems
4
  26.7%
10
  29.4%
Self-care: Moderate problems
4
  26.7%
7
  20.6%
Self-care: Severe problems
0
   0.0%
3
   8.8%
Self-care: Unable to do
3
  20.0%
4
  11.8%
Self-care: Data missing
1
   6.7%
4
  11.8%
Usual activities: No problems
8
  53.3%
17
  50.0%
Usual activities: Slight problems
3
  20.0%
5
  14.7%
Usual activities: Moderate problems
2
  13.3%
6
  17.6%
Usual activities: Severe problems
0
   0.0%
1
   2.9%
Usual activities: Unable to do
1
   6.7%
1
   2.9%
Usual activities: Data missing
1
   6.7%
4
  11.8%
Pain/discomfort: No problems
6
  40.0%
20
  58.8%
Pain/discomfort: Slight problems
8
  53.3%
7
  20.6%
Pain/discomfort: Moderate problems
0
   0.0%
3
   8.8%
Pain/discomfort: Severe problems
0
   0.0%
0
   0.0%
Pain/discomfort: Extreme problems
0
   0.0%
0
   0.0%
Pain/discomfort: Data missing
1
   6.7%
4
  11.8%
Anxiety/depression: No problems
11
  73.3%
22
  64.7%
Anxiety/depression: Slight problems
3
  20.0%
6
  17.6%
Anxiety/depression: Moderate problems
0
   0.0%
1
   2.9%
Anxiety/depression: Severe problems
0
   0.0%
1
   2.9%
Anxiety/depression: Extreme problems
0
   0.0%
0
   0.0%
Anxiety/depression: Data missing
1
   6.7%
4
  11.8%
26.Secondary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Intrathecal Drug Delivery Device (IDDD)-Related Adverse Events
Hide Description An adverse event (AE) was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational product-related. Treatment-emergent AEs for the no IT treatment group were defined as all AEs occurring on or after the date of randomization and at or before the end of the study (EOS) visit. Treatment-emergent AEs for the IT treatment group were defined as all AEs occurring on or after the date of the first IDDD implant surgery or Treatment-Emergentfirst dose of the investigational product (whichever was earlier) and at or before the EOS visit (+30 days) or 2 weeks after the removal of the last IDDD (whichever was later).
Time Frame From start of study treatment up to Week 53
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population included all randomized participants with any post-randomization safety assessments, analyzed according to the treatment received.
Arm/Group Title No IT Treatment IT Treatment
Hide Arm/Group Description:
Participants aged 3 to < 18 years received Elaprase therapy as standard of care for 12 months.
Participants aged 3 to < 18 years received IT injections of 10 mg Idursulfase-IT once monthly for 12 months through SOPH-A-PORT Mini S IDDD along with Elaprase.
Overall Number of Participants Analyzed 15 33
Measure Type: Count of Participants
Unit of Measure: Participants
TEAEs
14
  93.3%
33
 100.0%
IDDD-related AE
0
   0.0%
22
  66.7%
27.Secondary Outcome
Title Composite Scores of Bayley Scales of Infant Development (BSID-III) Scale in Substudy Population
Hide Description Participants who were younger than 3 years were assessed using the BSID-III. The BSID-­III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. The composite score for the cognitive scale, language scale, and motor scale are normed and have a mean=100, SD=15 and range of 40-160. Higher values denote stronger skills and abilities in the domain, indicating better outcomes. Participant wise data at evaluable timepoints was reported for this outcome.
Time Frame Baseline up to Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Substudy population included all participants enrolled and treated with investigational product in the substudy.
Arm/Group Title Substudy
Hide Arm/Group Description:
Participants aged less than 3 years received Idursulfase-IT monthly once for 12 months along with Elaprase at a dose of 5 mg if aged less than or equal to (<=) 8 months; 7.5 mg if aged greater than (>) 8 to 30 months and 10 mg if aged > 30 months to 3 years.
Overall Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: Score on a scale
Baseline: Participant 1: Motor Number Analyzed 1 participants
97
Week 16: Participant 1: Motor Number Analyzed 1 participants
82
Week 28: Participant 1: Motor Number Analyzed 1 participants
67
Week 40: Participant 1: Motor Number Analyzed 1 participants
94
Week 52: Participant 1: Motor Number Analyzed 1 participants
79
Baseline: Participant 2: Motor Number Analyzed 1 participants
82
Week 16: Participant 2: Motor Number Analyzed 1 participants
76
Week 28: Participant 2: Motor Number Analyzed 1 participants
82
Week 40: Participant 2: Motor Number Analyzed 1 participants
76
Baseline: Participant 3: Motor Number Analyzed 1 participants
82
Week 16: Participant 3: Motor Number Analyzed 1 participants
79
Week 28: Participant 3: Motor Number Analyzed 1 participants
88
Week 40: Participant 3: Motor Number Analyzed 1 participants
88
Week 52: Participant 3: Motor Number Analyzed 1 participants
82
Baseline: Participant 4: Motor Number Analyzed 1 participants
76
Week 16: Participant 4: Motor Number Analyzed 1 participants
91
Week 28: Participant 4: Motor Number Analyzed 1 participants
91
Baseline: Participant 5: Motor Number Analyzed 1 participants
94
Week 16: Participant 5: Motor Number Analyzed 1 participants
67
Week 28: Participant 5: Motor Number Analyzed 1 participants
85
Baseline: Participant 6: Motor Number Analyzed 1 participants
103
Week 16: Participant 6: Motor Number Analyzed 1 participants
107
Week 28: Participant 6: Motor Number Analyzed 1 participants
103
Week 40: Participant 6: Motor Number Analyzed 1 participants
107
Week 52: Participant 6: Motor Number Analyzed 1 participants
85
Baseline: Participant 7: Motor Number Analyzed 1 participants
85
Week 16: Participant 7: Motor Number Analyzed 1 participants
76
Week 28: Participant 7: Motor Number Analyzed 1 participants
79
Week 40: Participant 7: Motor Number Analyzed 1 participants
79
Baseline: Participant 8: Motor Number Analyzed 1 participants
91
Week 16: Participant 8: Motor Number Analyzed 1 participants
91
Week 28: Participant 8: Motor Number Analyzed 1 participants
88
Baseline: Participant 9: Motor Number Analyzed 1 participants
85
Week 16: Participant 9: Motor Number Analyzed 1 participants
88
Week 28:Participant 9: Motor Number Analyzed 1 participants
85
Week 40: Participant 9: Motor Number Analyzed 1 participants
85
Week 52: Participant 9: Motor Number Analyzed 1 participants
91
Baseline: Participant 1: Language Number Analyzed 1 participants
91
Week 16: Participant 1: Language Number Analyzed 1 participants
89
Week 28: Participant 1: Language Number Analyzed 1 participants
91
Week 40: Participant 1: Language Number Analyzed 1 participants
91
Week 52: Participant 1: Language Number Analyzed 1 participants
94
Baseline: Participant 2: Language Number Analyzed 1 participants
74
Week 16: Participant 2: Language Number Analyzed 1 participants
77
Week 28: Participant 2: Language Number Analyzed 1 participants
74
Week 40: Participant 2: Language Number Analyzed 1 participants
79
Baseline: Participant 3: Language Number Analyzed 1 participants
79
Week 16: Participant 3: Language Number Analyzed 1 participants
77
Week 28: Participant 3: Language Number Analyzed 1 participants
71
Week 40: Participant 3: Language Number Analyzed 1 participants
91
Week 52: Participant 3: Language Number Analyzed 1 participants
86
Baseline: Participant 4: Language Number Analyzed 1 participants
79
Week 16: Participant 4: Language Number Analyzed 1 participants
89
Week 28: Participant 4: Language Number Analyzed 1 participants
89
Baseline: Participant 5: Language Number Analyzed 1 participants
59
Week 16: Participant 5: Language Number Analyzed 1 participants
53
Week 28: Participant 5: Language Number Analyzed 1 participants
47
Baseline: Participant 6: Language Number Analyzed 1 participants
89
Week 16: Participant 6: Language Number Analyzed 1 participants
86
Week 28: Participant 6: Language Number Analyzed 1 participants
77
Week 40: Participant 6: Language Number Analyzed 1 participants
89
Week 52: Participant 6: Language Number Analyzed 1 participants
83
Baseline: Participant 7: Language Number Analyzed 1 participants
79
Week 16: Participant 7: Language Number Analyzed 1 participants
74
Week 28: Participant 7: Language Number Analyzed 1 participants
74
Week 40: Participant 7: Language Number Analyzed 1 participants
71
Baseline: Participant 8: Language Number Analyzed 1 participants
86
Week 16: Participant 8: Language Number Analyzed 1 participants
100
Week 28: Participant 8: Language Number Analyzed 1 participants
103
Baseline: Participant 9: Language Number Analyzed 1 participants
62
Week 16: Participant 9: Language Number Analyzed 1 participants
71
Week 28: Participant 9: Language Number Analyzed 1 participants
71
Week 40: Participant 9: Language Number Analyzed 1 participants
79
Week 52: Participant 9: Language Number Analyzed 1 participants
74
Baseline: Participant 1: Cognitive Number Analyzed 1 participants
85
Week 16: Participant 1: Cognitive Number Analyzed 1 participants
90
Week 28: Participant 1: Cognitive Number Analyzed 1 participants
90
Week 40: Participant 1: Cognitive Number Analyzed 1 participants
100
Week 52: Participant 1: Cognitive Number Analyzed 1 participants
95
Baseline: Participant 2: Cognitive Number Analyzed 1 participants
75
Week 16: Participant 2: Cognitive Number Analyzed 1 participants
90
Week 28: Participant 2: Cognitive Number Analyzed 1 participants
90
Week 40: Participant 2: Cognitive Number Analyzed 1 participants
85
Baseline: Participant 3: Cognitive Number Analyzed 1 participants
90
Week 16: Participant 3: Cognitive Number Analyzed 1 participants
85
Week 28: Participant 3: Cognitive Number Analyzed 1 participants
90
Week 40: Participant 3: Cognitive Number Analyzed 1 participants
90
Week 52: Participant 3: Cognitive Number Analyzed 1 participants
85
Baseline: Participant 4: Cognitive Number Analyzed 1 participants
80
Week 16: Participant 4: Cognitive Number Analyzed 1 participants
95
Week 28: Participant 4: Cognitive Number Analyzed 1 participants
100
Baseline: Participant 5: Cognitive Number Analyzed 1 participants
85
Week 16: Participant 5: Cognitive Number Analyzed 1 participants
85
Week 28: Participant 5: Cognitive Number Analyzed 1 participants
80
Baseline: Participant 6: Cognitive Number Analyzed 1 participants
80
Week 16: Participant 6: Cognitive Number Analyzed 1 participants
100
Week 28: Participant 6: Cognitive Number Analyzed 1 participants
95
Week 40: Participant 6: Cognitive Number Analyzed 1 participants
105
Week 52: Participant 6: Cognitive Number Analyzed 1 participants
90
Baseline: Participant 7: Cognitive Number Analyzed 1 participants
85
Week 16: Participant 7: Cognitive Number Analyzed 1 participants
90
Week 28: Participant 7: Cognitive Number Analyzed 1 participants
85
Week 40: Participant 7: Cognitive Number Analyzed 1 participants
80
Baseline: Participant 8: Cognitive Number Analyzed 1 participants
90
Week 16: Participant 8: Cognitive Number Analyzed 1 participants
100
Week 28: Participant 8: Cognitive Number Analyzed 1 participants
110
Baseline: Participant 9: Cognitive Number Analyzed 1 participants
85
Week 16: Participant 9: Cognitive Number Analyzed 1 participants
90
Week 28: Participant 9: Cognitive Number Analyzed 1 participants
90
Week 40: Participant 9: Cognitive Number Analyzed 1 participants
90
Week 52: Participant 9: Cognitive Number Analyzed 1 participants
95
28.Secondary Outcome
Title Percentile Scores of Bayley Scales of Infant Development (BSID-III) Scale in Substudy Population
Hide Description Participants who were younger than 3 years were assessed using the BSID-III. The BSID-­III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. Percentile scores range from 1 to 99 with 50 as the mean and median. Higher percentile means higher the rank of the child relative to the normed population. Participant wise data at evaluable timepoints was reported for this outcome.
Time Frame Baseline up to Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Substudy population included all participants enrolled and treated with investigational product in the substudy.
Arm/Group Title Substudy
Hide Arm/Group Description:
Participants aged less than 3 years received Idursulfase-IT monthly once for 12 months along with Elaprase at a dose of 5 mg if aged less than or equal to (<=) 8 months; 7.5 mg if aged greater than (>) 8 to 30 months and 10 mg if aged > 30 months to 3 years.
Overall Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: Percentile score
Baseline: Participant 1: Motor Number Analyzed 1 participants
42
Week 16: Participant 1: Motor Number Analyzed 1 participants
12
Week 28: Participant 1: Motor Number Analyzed 1 participants
1
Week 40: Participant 1: Motor Number Analyzed 1 participants
34
Week 52: Participant 1: Motor Number Analyzed 1 participants
8
Baseline: Participant 2: Motor Number Analyzed 1 participants
12
Week 16: Participant 2: Motor Number Analyzed 1 participants
5
Week 28: Participant 2: Motor Number Analyzed 1 participants
12
Week 40: Participant 2: Motor Number Analyzed 1 participants
5
Baseline: Participant 3: Motor Number Analyzed 1 participants
12
Week 16: Participant 3: Motor Number Analyzed 1 participants
8
Week 28: Participant 3: Motor Number Analyzed 1 participants
21
Week 40: Participant 3: Motor Number Analyzed 1 participants
21
Week 52: Participant 3: Motor Number Analyzed 1 participants
12
Baseline: Participant 4: Motor Number Analyzed 1 participants
5
Week 16: Participant 4: Motor Number Analyzed 1 participants
27
Week 28: Participant 4: Motor Number Analyzed 1 participants
27
Baseline: Participant 5: Motor Number Analyzed 1 participants
34
Week 16: Participant 5: Motor Number Analyzed 1 participants
1
Week 28: Participant 5: Motor Number Analyzed 1 participants
16
Baseline: Participant 6: Motor Number Analyzed 1 participants
58
Week 16: Participant 6: Motor Number Analyzed 1 participants
68
Week 28: Participant 6: Motor Number Analyzed 1 participants
58
Week 40: Participant 6: Motor Number Analyzed 1 participants
68
Week 52: Participant 6: Motor Number Analyzed 1 participants
16
Baseline: Participant 7: Motor Number Analyzed 1 participants
16
Week 16: Participant 7: Motor Number Analyzed 1 participants
5
Week 28: Participant 7: Motor Number Analyzed 1 participants
8
Week 40: Participant 7: Motor Number Analyzed 1 participants
1
Baseline: Participant 8: Motor Number Analyzed 1 participants
27
Week 16: Participant 8: Motor Number Analyzed 1 participants
27
Week 28: Participant 8: Motor Number Analyzed 1 participants
21
Baseline: Participant 9: Motor Number Analyzed 1 participants
16
Week 16: Participant 9: Motor Number Analyzed 1 participants
21
Week 28: Participant 9: Motor Number Analyzed 1 participants
16
Week 40: Participant 9: Motor Number Analyzed 1 participants
16
Week 52: Participant 9: Motor Number Analyzed 1 participants
27
Baseline: Participant 1: Language Number Analyzed 1 participants
27
Week 16: Participant 1: Language Number Analyzed 1 participants
23
Week 28: Participant 1: Language Number Analyzed 1 participants
27
Week 40: Participant 1: Language Number Analyzed 1 participants
27
Week 52: Participant 1: Language Number Analyzed 1 participants
34
Baseline: Participant 2: Language Number Analyzed 1 participants
4
Week 16: Participant 2: Language Number Analyzed 1 participants
6
Week 28: Participant 2: Language Number Analyzed 1 participants
4
Week 40: Participant 2: Language Number Analyzed 1 participants
8
Baseline: Participant 3: Language Number Analyzed 1 participants
8
Week 16: Participant 3: Language Number Analyzed 1 participants
6
Week 28: Participant 3: Language Number Analyzed 1 participants
3
Week 40: Participant 3: Language Number Analyzed 1 participants
27
Week 52: Participant 3: Language Number Analyzed 1 participants
18
Baseline: Participant 4: Language Number Analyzed 1 participants
8
Week 16: Participant 4: Language Number Analyzed 1 participants
23
Week 28: Participant 4: Language Number Analyzed 1 participants
23
Baseline: Participant 5: Language Number Analyzed 1 participants
0.3
Week 16: Participant 5: Language Number Analyzed 1 participants
0.1
Week 28: Participant 5: Language Number Analyzed 1 participants
0.1
Baseline: Participant 6: Language Number Analyzed 1 participants
23
Week 16: Participant 6: Language Number Analyzed 1 participants
18
Week 28: Participant 6: Language Number Analyzed 1 participants
6
Week 40: Participant 6: Language Number Analyzed 1 participants
23
Week 52: Participant 6: Language Number Analyzed 1 participants
13
Baseline: Participant 7: Language Number Analyzed 1 participants
8
Week 16: Participant 7: Language Number Analyzed 1 participants
4
Week 28: Participant 7: Language Number Analyzed 1 participants
4
Week 40: Participant 7: Language Number Analyzed 1 participants
3
Baseline: Participant 8: Language Number Analyzed 1 participants
18
Week 16: Participant 8: Language Number Analyzed 1 participants
50
Week 28: Participant 8: Language Number Analyzed 1 participants
58
Baseline: Participant 9: Language Number Analyzed 1 participants
1
Week 16: Participant 9: Language Number Analyzed 1 participants
3
Week 28: Participant 9: Language Number Analyzed 1 participants
3
Week 40: Participant 9: Language Number Analyzed 1 participants
8
Week 52: Participant 9: Language Number Analyzed 1 participants
4
Baseline: Participant 1: Cognitive Number Analyzed 1 participants
16
Week 16: Participant 1: Cognitive Number Analyzed 1 participants
25
Week 28: Participant 1: Cognitive Number Analyzed 1 participants
25
Week 40: Participant 1: Cognitive Number Analyzed 1 participants
50
Week 52: Participant 1: Cognitive Number Analyzed 1 participants
37
Baseline: Participant 2: Cognitive Number Analyzed 1 participants
5
Week 16: Participant 2: Cognitive Number Analyzed 1 participants
25
Week 28: Participant 2: Cognitive Number Analyzed 1 participants
25
Week 40: Participant 2: Cognitive Number Analyzed 1 participants
16
Baseline: Participant 3: Cognitive Number Analyzed 1 participants
25
Week 16: Participant 3: Cognitive Number Analyzed 1 participants
16
Week 28: Participant 3: Cognitive Number Analyzed 1 participants
25
Week 40: Participant 3: Cognitive Number Analyzed 1 participants
25
Week 52: Participant 3: Cognitive Number Analyzed 1 participants
16
Baseline: Participant 4: Cognitive Number Analyzed 1 participants
9
Week 16: Participant 4: Cognitive Number Analyzed 1 participants
37
Week 28: Participant 4: Cognitive Number Analyzed 1 participants
50
Baseline: Participant 5: Cognitive Number Analyzed 1 participants
16
Week 16: Participant 5: Cognitive Number Analyzed 1 participants
16
Week 28: Participant 5: Cognitive Number Analyzed 1 participants
9
Baseline: Participant 6: Cognitive Number Analyzed 1 participants
9
Week 16: Participant 6: Cognitive Number Analyzed 1 participants
50
Week 28: Participant 6: Cognitive Number Analyzed 1 participants
37
Week 40: Participant 6: Cognitive Number Analyzed 1 participants
63
Week 52: Participant 6: Cognitive Number Analyzed 1 participants
25
Baseline: Participant 7: Cognitive Number Analyzed 1 participants
16
Week 16: Participant 7: Cognitive Number Analyzed 1 participants
25
Week 28: Participant 7: Cognitive Number Analyzed 1 participants
16
Week 40: Participant 7: Cognitive Number Analyzed 1 participants
9
Baseline: Participant 8: Cognitive Number Analyzed 1 participants
25
Week 16: Participant 8: Cognitive Number Analyzed 1 participants
50
Week 28: Participant 8: Cognitive Number Analyzed 1 participants
75
Baseline: Participant 9: Cognitive Number Analyzed 1 participants
16
Week 16: Participant 9: Cognitive Number Analyzed 1 participants
25
Week 28: Participant 9: Cognitive Number Analyzed 1 participants
25
Week 40: Participant 9: Cognitive Number Analyzed 1 participants
25
Week 52: Participant 9: Cognitive Number Analyzed 1 participants
37
29.Secondary Outcome
Title Age Equivalent Scores of Bayley Scales of Infant Development (BSID-III) Scale in Substudy Population
Hide Description Participants who were younger than 3 years were assessed using the BSID-III. The BSID-­III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. Standardized scores (range 40-160) were converted to age­ equivalent scores to measure ability, skill, and knowledge expressed as the age at which most individuals reach the same level (age norm; range: 0, unbound). Higher values present better outcomes. Participant wise data at evaluable timepoints was reported for this outcome.
Time Frame Baseline up to Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Substudy population included all participants enrolled and treated with investigational product in the substudy.
Arm/Group Title Substudy
Hide Arm/Group Description:
Participants aged less than 3 years received Idursulfase-IT monthly once for 12 months along with Elaprase at a dose of 5 mg if aged less than or equal to (<=) 8 months; 7.5 mg if aged greater than (>) 8 to 30 months and 10 mg if aged > 30 months to 3 years.
Overall Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: Score on a scale
Baseline: Participant 1: Motor-Fine Number Analyzed 1 participants
28
Baseline: Participant 1: Motor-Gross Number Analyzed 1 participants
24
Week 16: Participant 1: Motor-Fine Number Analyzed 1 participants
28
Week 16: Participant 1: Motor-Gross Number Analyzed 1 participants
20
Week 28: Participant 1: Motor-Fine Number Analyzed 1 participants
18
Week 28: Participant 1: Motor-Gross Number Analyzed 1 participants
21
Week 40: Participant 1: Motor-Fine Number Analyzed 1 participants
32
Week 40: Participant 1: Motor-Gross Number Analyzed 1 participants
38
Week 52: Participant 1: Motor-Fine Number Analyzed 1 participants
34
Week 52: Participant 1: Motor-Gross Number Analyzed 1 participants
24
Baseline: Participant 2: Motor-Fine Number Analyzed 1 participants
25
Baseline: Participant 2: Motor-Gross Number Analyzed 1 participants
20
Week 16: Participant 2: Motor-Fine Number Analyzed 1 participants
28
Week 16: Participant 2: Motor-Gross Number Analyzed 1 participants
18
Week 28: Participant 2: Motor-Fine Number Analyzed 1 participants
32
Week 28: Participant 2: Motor-Gross Number Analyzed 1 participants
24
Week 40: Participant 2: Motor-Fine Number Analyzed 1 participants
29
Week 40: Participant 2: Motor-Gross Number Analyzed 1 participants
23
Baseline: Participant 3: Motor-Fine Number Analyzed 1 participants
26
Baseline: Participant 3: Motor-Gross Number Analyzed 1 participants
18
Week 16: Participant 3: Motor-Fine Number Analyzed 1 participants
26
Week 16: Participant 3: Motor-Gross Number Analyzed 1 participants
21
Week 28: Participant 3: Motor-Fine Number Analyzed 1 participants
32
Week 28: Participant 3: Motor-Gross Number Analyzed 1 participants
31
Week 40: Participant 3: Motor-Fine Number Analyzed 1 participants
32
Week 40: Participant 3: Motor-Gross Number Analyzed 1 participants
29
Week 52: Participant 3: Motor-Fine Number Analyzed 1 participants
29
Week 52: Participant 3: Motor-Gross Number Analyzed 1 participants
29
Baseline: Participant 4: Motor-Fine Number Analyzed 1 participants
29
Baseline: Participant 4: Motor-Gross Number Analyzed 1 participants
19
Week 16: Participant 4: Motor-Fine Number Analyzed 1 participants
33
Week 16: Participant 4: Motor-Gross Number Analyzed 1 participants
38
Week 28: Participant 4: Motor-Fine Number Analyzed 1 participants
31
Week 28: Participant 4: Motor-Gross Number Analyzed 1 participants
41
Baseline: Participant 5: Motor-Fine Number Analyzed 1 participants
28
Baseline: Participant 5: Motor-Gross Number Analyzed 1 participants
27
Week 16: Participant 5: Motor-Fine Number Analyzed 1 participants
25
Week 16: Participant 5: Motor-Gross Number Analyzed 1 participants
20
Week 28: Participant 5: Motor-Fine Number Analyzed 1 participants
29
Week 28: Participant 5: Motor-Gross Number Analyzed 1 participants
31
Week 40: Participant 5: Motor-Fine Number Analyzed 1 participants
26
Week 40: Participant 5: Motor-Gross Number Analyzed 1 participants
27
Week 52: Participant 5: Motor-Fine Number Analyzed 1 participants
28
Week 52: Participant 5: Motor-Gross Number Analyzed 1 participants
35
Baseline: Participant 6: Motor-Fine Number Analyzed 1 participants
16
Baseline: Participant 6: Motor-Gross Number Analyzed 1 participants
15
Week 16: Participant 6: Motor-Fine Number Analyzed 1 participants
25
Week 16: Participant 6: Motor-Gross Number Analyzed 1 participants
20
Week 28: Participant 6: Motor-Fine Number Analyzed 1 participants
26
Week 28: Participant 6: Motor-Gross Number Analyzed 1 participants
23
Week 40: Participant 6: Motor-Fine Number Analyzed 1 participants
31
Week 40: Participant 6: Motor-Gross Number Analyzed 1 participants
26
Week 52: Participant 6: Motor-Fine Number Analyzed 1 participants
25
Week 52: Participant 6: Motor-Gross Number Analyzed 1 participants
20
Baseline: Participant 7: Motor-Fine Number Analyzed 1 participants
26
Baseline: Participant 7: Motor-Gross Number Analyzed 1 participants
20
Week 16: Participant 7: Motor-Fine Number Analyzed 1 participants
25
Week 16: Participant 7: Motor-Gross Number Analyzed 1 participants
23
Week 28: Participant 7: Motor-Fine Number Analyzed 1 participants
28
Week 28: Participant 7: Motor-Gross Number Analyzed 1 participants
24
Week 40: Participant 7: Motor-Fine Number Analyzed 1 participants
29
Week 40: Participant 7: Motor-Gross Number Analyzed 1 participants
27
Week 52: Participant 7: Motor-Fine Number Analyzed 1 participants
29
Week 52: Participant 7: Motor-Gross Number Analyzed 1 participants
31
Baseline: Participant 8: Motor-Fine Number Analyzed 1 participants
31
Baseline: Participant 8: Motor-Gross Number Analyzed 1 participants
31
Week 16: Participant 8: Motor-Fine Number Analyzed 1 participants
38
Week 16: Participant 8: Motor-Gross Number Analyzed 1 participants
31
Week 28: Participant 8: Motor-Fine Number Analyzed 1 participants
35
Week 28: Participant 8: Motor-Gross Number Analyzed 1 participants
31
Baseline: Participant 9: Motor-Fine Number Analyzed 1 participants
25
Baseline: Participant 9: Motor-Gross Number Analyzed 1 participants
18
Week 16: Participant 9: Motor-Fine Number Analyzed 1 participants
27
Week 16: Participant 9: Motor-Gross Number Analyzed 1 participants
21
Week 28: Participant 9: Motor-Fine Number Analyzed 1 participants
31
Week 28: Participant 9: Motor-Gross Number Analyzed 1 participants
20
Week 40: Participant 9: Motor-Fine Number Analyzed 1 participants
31
Week 40: Participant 9: Motor-Gross Number Analyzed 1 participants
26
Week 52: Participant 9: Motor-Fine Number Analyzed 1 participants
34
Week 52: Participant 9: Motor-Gross Number Analyzed 1 participants
31
Baseline: Participant 1: Language-Receptive Number Analyzed 1 participants
22
Baseline: Participant 1: Language-Expressive Number Analyzed 1 participants
25
Week 16: Participant 1: Language-Receptive Number Analyzed 1 participants
24
Week 16: Participant 1: Language-Expressive Number Analyzed 1 participants
27
Week 28: Participant 1: Language-Receptive Number Analyzed 1 participants
26
Week 28: Participant 1: Language-Expressive Number Analyzed 1 participants
33
Week 40: Participant 1: Language-Receptive Number Analyzed 1 participants
30
Week 40: Participant 1: Language-Expressive Number Analyzed 1 participants
33
Week 52: Participant 1: Language-Receptive Number Analyzed 1 participants
32
Week 52: Participant 1: Language-Expressive Number Analyzed 1 participants
34
Baseline: Participant 2: Language-Receptive Number Analyzed 1 participants
19
Baseline: Participant 2: Language-Expressive Number Analyzed 1 participants
18
Week 16: Participant 2: Language-Receptive Number Analyzed 1 participants
23
Week 16: Participant 2: Language-Expressive Number Analyzed 1 participants
19
Week 28: Participant 2: Language-Receptive Number Analyzed 1 participants
20
Week 28: Participant 2: Language-Expressive Number Analyzed 1 participants
24
Week 40: Participant 2: Language-Receptive Number Analyzed 1 participants
26
Week 40: Participant 2: Language-Expressive Number Analyzed 1 participants
25
Baseline: Participant 3: Language-Receptive Number Analyzed 1 participants
19
Baseline: Participant 3: Language-Expressive Number Analyzed 1 participants
19
Week 16: Participant 3: Language-Receptive Number Analyzed 1 participants
19
Week 16: Participant 3: Language-Expressive Number Analyzed 1 participants
23
Week 28: Participant 3: Language-Receptive Number Analyzed 1 participants
21
Week 28: Participant 3: Language-Expressive Number Analyzed 1 participants
22
Week 40: Participant 3: Language-Receptive Number Analyzed 1 participants
29
Week 40: Participant 3: Language-Expressive Number Analyzed 1 participants
33
Week 52: Participant 3: Language-Receptive Number Analyzed 1 participants
29
Week 52: Participant 3: Language-Expressive Number Analyzed 1 participants
29
Baseline: Participant 4: Language-Receptive Number Analyzed 1 participants
21
Baseline: Participant 4: Language-Expressive Number Analyzed 1 participants
25
Week 16: Participant 4: Language-Receptive Number Analyzed 1 participants
32
Week 16: Participant 4: Language-Expressive Number Analyzed 1 participants
30
Week 28: Participant 4: Language-Receptive Number Analyzed 1 participants
28
Week 28: Participant 4: Language-Expressive Number Analyzed 1 participants
38
Baseline: Participant 5: Language-Receptive Number Analyzed 1 participants
11
Baseline: Participant 5: Language-Expressive Number Analyzed 1 participants
12
Week 16: Participant 5: Language-Receptive Number Analyzed 1 participants
11
Week 16: Participant 5: Language-Expressive Number Analyzed 1 participants
7
Week 28: Participant 5: Language-Receptive Number Analyzed 1 participants
0.52
Week 28: Participant 5: Language-Expressive Number Analyzed 1 participants
7
Week 40: Participant 5: Language-Receptive Number Analyzed 1 participants
11
Week 40: Participant 5: Language-Expressive Number Analyzed 1 participants
13
Week 52: Participant 5: Language-Receptive Number Analyzed 1 participants
11
Week 52: Participant 5: Language-Expressive Number Analyzed 1 participants
10
Baseline: Participant 6: Language-Receptive Number Analyzed 1 participants
14
Baseline: Participant 6: Language-Expressive Number Analyzed 1 participants
12
Week 16: Participant 6: Language-Receptive Number Analyzed 1 participants
19
Week 16: Participant 6: Language-Expressive Number Analyzed 1 participants
16
Week 28: Participant 6: Language-Receptive Number Analyzed 1 participants
19
Week 28: Participant 6: Language-Expressive Number Analyzed 1 participants
14
Week 40: Participant 6: Language-Receptive Number Analyzed 1 participants
25
Week 40: Participant 6: Language-Expressive Number Analyzed 1 participants
19
Week 52: Participant 6: Language-Receptive Number Analyzed 1 participants
24
Week 52: Participant 6: Language-Expressive Number Analyzed 1 participants
19
Baseline: Participant 7: Language-Receptive Number Analyzed 1 participants
19
Baseline: Participant 7: Language-Expressive Number Analyzed 1 participants
21
Week 16: Participant 7: Language-Receptive Number Analyzed 1 participants
16
Week 16: Participant 7: Language-Expressive Number Analyzed 1 participants
23
Week 28: Participant 7: Language-Receptive Number Analyzed 1 participants
18
Week 28: Participant 7: Language-Expressive Number Analyzed 1 participants
25
Week 40: Participant 7: Language-Receptive Number Analyzed 1 participants
18
Week 40: Participant 7: Language-Expressive Number Analyzed 1 participants
24
Week 52: Participant 7: Language-Receptive Number Analyzed 1 participants
19
Week 52: Participant 7: Language-Expressive Number Analyzed 1 participants
22
Baseline: Participant 8: Language-Receptive Number Analyzed 1 participants
29
Baseline: Participant 8: Language-Expressive Number Analyzed 1 participants
26
Week 16: Participant 8: Language-Receptive Number Analyzed 1 participants
42
Week 16: Participant 8: Language-Expressive Number Analyzed 1 participants
38
Week 28: Participant 8: Language-Receptive Number Analyzed 1 participants
42
Week 28: Participant 8: Language-Expressive Number Analyzed 1 participants
34
Baseline: Participant 9: Language-Receptive Number Analyzed 1 participants
18
Baseline: Participant 9: Language-Expressive Number Analyzed 1 participants
5
Week 16: Participant 9: Language-Receptive Number Analyzed 1 participants
25
Week 16: Participant 9: Language-Expressive Number Analyzed 1 participants
2
Week 28: Participant 9: Language-Receptive Number Analyzed 1 participants
28
Week 28: Participant 9: Language-Expressive Number Analyzed 1 participants
7
Week 40: Participant 9: Language-Receptive Number Analyzed 1 participants
35
Week 40: Participant 9: Language-Expressive Number Analyzed 1 participants
16
Week 52: Participant 9: Language-Receptive Number Analyzed 1 participants
30
Week 52: Participant 9: Language-Expressive Number Analyzed 1 participants
17
Baseline: Participant 1: Cognitive Number Analyzed 1 participants
22
Week 16: Participant 1: Cognitive Number Analyzed 1 participants
26
Week 28: Participant 1: Cognitive Number Analyzed 1 participants
30
Week 40: Participant 1: Cognitive Number Analyzed 1 participants
38
Week 52: Participant 1: Cognitive Number Analyzed 1 participants
36
Baseline: Participant 2: Cognitive Number Analyzed 1 participants
21
Week 16: Participant 2: Cognitive Number Analyzed 1 participants
28
Week 28: Participant 2: Cognitive Number Analyzed 1 participants
29
Week 40: Participant 2: Cognitive Number Analyzed 1 participants
28
Baseline: Participant 3: Cognitive Number Analyzed 1 participants
24
Week 16: Participant 3: Cognitive Number Analyzed 1 participants
25
Week 28: Participant 3: Cognitive Number Analyzed 1 participants
28
Week 40: Participant 3: Cognitive Number Analyzed 1 participants
28
Week 52: Participant 3: Cognitive Number Analyzed 1 participants
29
Baseline: Participant 4: Cognitive Number Analyzed 1 participants
23
Week 16: Participant 4: Cognitive Number Analyzed 1 participants
36
Week 28: Participant 4: Cognitive Number Analyzed 1 participants
41
Baseline: Participant 5: Cognitive Number Analyzed 1 participants
23
Week 16: Participant 5: Cognitive Number Analyzed 1 participants
27
Week 28: Participant 5: Cognitive Number Analyzed 1 participants
25
Week 40: Participant 5: Cognitive Number Analyzed 1 participants
28
Week 52: Participant 5: Cognitive Number Analyzed 1 participants
28
Baseline: Participant 6: Cognitive Number Analyzed 1 participants
12
Week 16: Participant 6: Cognitive Number Analyzed 1 participants