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Trial record 1 of 1 for:    aglu03306
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High Dose or High Dose Frequency Study of Alglucosidase Alfa

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00483379
Recruitment Status : Completed
First Posted : June 7, 2007
Results First Posted : April 22, 2011
Last Update Posted : March 7, 2014
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Pompe Disease
Glycogen Storage Disease Type II (GSD-II)
Glycogenesis 2 Acid Maltase Deficiency
Intervention Biological: alglucosidase alfa
Enrollment 13
Recruitment Details  
Pre-assignment Details Fourteen participants were screened and enrolled; however, one withdrew before receiving any study infusions due to the burden of weekly trips to the medical center.
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm. Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Period Title: Treatment Period
Started 6 7
Completed 4 7
Not Completed 2 0
Reason Not Completed
Adverse Event             1             0
Withdrawal by Subject             1             0
Period Title: Extension Period
Started 1 [1] 2 [1]
Completed 1 2
Not Completed 0 0
[1]
Extension allowed late-onset participants access to treatment until commercially available.
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week Total
Hide Arm/Group Description Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm. Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm. Total of all reporting groups
Overall Number of Baseline Participants 6 7 13
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants 7 participants 13 participants
23.3  (27.75) 16.8  (15.56) 19.8  (21.29)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 6 participants 7 participants 13 participants
<18 years 4 5 9
>= 18 and <=65 years 2 2 4
>65 years 0 0 0
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 7 participants 13 participants
Female
2
  33.3%
3
  42.9%
5
  38.5%
Male
4
  66.7%
4
  57.1%
8
  61.5%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 7 participants 13 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
1
  14.3%
1
   7.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
6
 100.0%
6
  85.7%
12
  92.3%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Life-stage of Disease Onset  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 6 participants 7 participants 13 participants
Infantile-onset Pompe Disease 4 5 9
Late-onset Pompe Disease 2 2 4
Parameter in Clinical Decline   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 6 participants 7 participants 13 participants
Cardiac 0 0 0
Respiratory 1 1 2
Motor Skills 5 6 11
[1]
Measure Description: Participant counts of the parameter in clinical decline (cardiac, respiratory or motor skills as compared to their condition prior to the beginning alglucosidase alfa treatment) for which participants were included in the study.
Cross-Reactive Immunologic Material (CRIM) Assay Result  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 6 participants 7 participants 13 participants
Positive 0 3 3
Negative 1 0 1
Unknown 5 4 9
1.Primary Outcome
Title Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment
Hide Description Participants were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each participant was evaluated at Week 52 for change from baseline in the criteria that declined; respiratory decline as measured by change in ventilator use is summarized in this outcome. Ventilator use might have improved (less use of ventilator support), had no change, or worsened (more use of ventilator support). Each participant served as his or her own control.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who enrolled due to decline in respiratory function while on standard treatment.
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 1 1
Measure Type: Number
Unit of Measure: participants
Improved 0 0
No change (on invasive ventilator for 24 hrs) 0 1
Worsened 0 0
Not evaluated 1 0
2.Primary Outcome
Title Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Motor Function Decline on Standard Treatment
Hide Description Participants were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each participant was evaluated at Week 52 for change from baseline in the criteria that declined; motor function decline primarily based on Gross Motor Function Measure 66 and Pompe Pediatric Evaluation of Disability Inventory results is summarized. Participants could gain motor function (improve), had no change (declined stopped), or continued loss (worsened). Each participant served as his or her own control.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who enrolled due to decline in motor function while on standard treatment.
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 5 6
Measure Type: Number
Unit of Measure: participants
Gained gross or fine motor skills 2 4
No change 1 2
Continued motor loss 1 0
Not evaluated 1 0
3.Primary Outcome
Title Summary of Participants Reporting Treatment-Emergent Adverse Events During the Treatment Period
Hide Description Overall safety summary of participants experiencing Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on Treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment.
Time Frame Day 1 up to Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population comprised of all participants who received intervention.
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 6 7
Measure Type: Number
Unit of Measure: participants
Participants with AEs 6 7
Related AEs 0 2
Not related AEs 6 7
Mild AEs 6 6
Moderate AEs 3 2
Severe AEs 2 0
AEs leading to discontinuation from study 1 0
Deaths 1 0
Infusion Associated Reactions 0 2
Serious AEs 2 1
4.Secondary Outcome
Title Baseline Values for Left Ventricular Mass (LVM) Z-Scores
Hide Description Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. Negative values indicate a smaller than mean LVM and values higher than 0 indicate a larger LVM than the mean. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children’s Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist.
Time Frame Day 0
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population of participants with LVM data
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 5 5
Median (Full Range)
Unit of Measure: Z-score
0.3
(-1.2 to 6.3)
-0.3
(-1.2 to 2.2)
5.Secondary Outcome
Title Change From Baseline in Left Ventricular Mass (LVM) Z-Score at Week 52
Hide Description Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children’s Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population of participants with LVM data at both timepoints
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: Z-score
0.3
(-0.8 to 1.2)
0.4
(-0.1 to 0.5)
6.Secondary Outcome
Title Baseline Values for Left Ventricular Mass Index (LVMI)
Hide Description Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline. Left Ventricular Mass is adjusted to the participant's body surface area in the calculation of LVMI.
Time Frame Day 0
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population of participants with LVMI data
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 5 5
Median (Full Range)
Unit of Measure: g/m^2
62.1
(49.0 to 187.3)
56.5
(45.4 to 73.1)
7.Secondary Outcome
Title Change From Baseline in Left Ventricular Mass Index (LVMI) at Week 52
Hide Description Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline and after 12 months of treatment (Week 52). Left Ventricular Mass is adjusted to the participant's body surface area in the calculation of LVMI.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population of participants with LVMI data at both timepoints
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: g/m^2
12.5
(-10.9 to 15.5)
4.0
(-5.3 to 5.8)
8.Secondary Outcome
Title Change From Baseline in Ventilator Use at Last Assessment (Approximately Week 52)
Hide Description The change from baseline in ventilator use at the last assessment is summarized as improved (less use of ventilator support), no change, worsened (increased use of ventilator support), and did not use ventilator support.
Time Frame Baseline, approximately Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population. The participant in the worsened category died after week 52.
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 6 7
Measure Type: Number
Unit of Measure: participants
Improved 0 0
No change 2 3
Worsened 1 0
Did not use ventilator 3 4
9.Secondary Outcome
Title Change From Baseline in Body Strength Measured by the Manual Muscle Testing (MMT) Total Score at Week 52
Hide Description Body strength is measured by the MMT score on a scale of 0-10 with higher scores representing greater body strength.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population of participants >= 8 years old. Due to the age restriction and small study population, the number of participants analyzed is too small for results to be meaningful.
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
10.Secondary Outcome
Title Baseline Values of Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results
Hide Description The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions.
Time Frame Day 0
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 6 6
Mean (Standard Deviation)
Unit of Measure: units on a scale
65.0  (60.52) 82.8  (84.00)
11.Secondary Outcome
Title Change From Baseline in Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results at Week 52
Hide Description The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 4 6
Mean (Standard Deviation)
Unit of Measure: units on a scale
6.0  (8.49) 6.7  (6.12)
12.Secondary Outcome
Title Baseline Values in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI)
Hide Description The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near "0" reflecting low capability and scores near "100" reflecting high capability.
Time Frame Day 0
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 6 7
Mean (Standard Deviation)
Unit of Measure: units on a scale
38.3  (20.94) 46.8  (21.26)
13.Secondary Outcome
Title Change From Baseline in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at Week 52
Hide Description The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Change from baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near “0” reflecting low capability and scores near “100” reflecting high capability.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 4 7
Mean (Standard Deviation)
Unit of Measure: units on a scale
0.6  (4.28) 3.5  (3.84)
14.Secondary Outcome
Title Baseline Values for Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36)
Hide Description Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for participants ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life.
Time Frame Day 0
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population of participants >= 14 years old.
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 2 3
Mean (Standard Deviation)
Unit of Measure: units on a scale
31.3  (2.84) 36.0  (9.33)
15.Secondary Outcome
Title Change From Baseline in Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36) at Week 52
Hide Description Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for participants ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population of participants >= 14 years old.
Arm/Group Title Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description:
Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
Overall Number of Participants Analyzed 1 3
Mean (Standard Deviation)
Unit of Measure: units on a scale
2.5 4.4  (11.24)
Time Frame Treatment period AEs were collected up to week 52. Extension period AEs were collected following completion of the treatment period until the product was commercially available (up to week 118).
Adverse Event Reporting Description

In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.

Events are listed independent of relationship to treatment reported.

 
Arm/Group Title Treatment: Alglucosidase Alfa 20 mg/kg Every Week Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week Extension: Alglucosidase Alfa 20 mg/kg Every Week Extension: Alglucosidase Alfa 40 mg/kg Every Other Week
Hide Arm/Group Description Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm. Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm. The extension period of the study allowed late-onset participants access to the same treatment they took in the treatment period until the product was commercially available. The extension period of the study allowed late-onset participants access to the same treatment they took in the treatment period until the product was commercially available.
All-Cause Mortality
Treatment: Alglucosidase Alfa 20 mg/kg Every Week Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week Extension: Alglucosidase Alfa 20 mg/kg Every Week Extension: Alglucosidase Alfa 40 mg/kg Every Other Week
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Treatment: Alglucosidase Alfa 20 mg/kg Every Week Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week Extension: Alglucosidase Alfa 20 mg/kg Every Week Extension: Alglucosidase Alfa 40 mg/kg Every Other Week
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/6 (33.33%)   1/7 (14.29%)   1/1 (100.00%)   0/2 (0.00%) 
Cardiac disorders         
Supraventricular tachycardia  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Gastrointestinal disorders         
Dysphagia  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Infections and infestations         
Device related infection  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Pneumonia  1  2/6 (33.33%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Injury, poisoning and procedural complications         
Fibula fracture  1  0/6 (0.00%)  0/7 (0.00%)  1/1 (100.00%)  0/2 (0.00%) 
Investigations         
Weight decreased  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Respiratory failure  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Treatment: Alglucosidase Alfa 20 mg/kg Every Week Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week Extension: Alglucosidase Alfa 20 mg/kg Every Week Extension: Alglucosidase Alfa 40 mg/kg Every Other Week
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   6/6 (100.00%)   7/7 (100.00%)   1/1 (100.00%)   1/2 (50.00%) 
Blood and lymphatic system disorders         
Lymphadenitis  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Lymphadenopathy  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Cardiac disorders         
Right ventricular hypertrophy  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Tachycardia  1  1/6 (16.67%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Ear and labyrinth disorders         
Hypoacusis  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Eye disorders         
Dry eye  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Eyelid ptosis  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Gastrointestinal disorders         
Abdominal discomfort  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Abdominal pain upper  1  2/6 (33.33%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Amalgam tattoo  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Anal fissure  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Aphthous stomatitis  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Constipation  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Diarrhoea  1  2/6 (33.33%)  2/7 (28.57%)  0/1 (0.00%)  0/2 (0.00%) 
Haematochezia  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Nausea  1  1/6 (16.67%)  0/7 (0.00%)  1/1 (100.00%)  0/2 (0.00%) 
Vomiting  1  2/6 (33.33%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
General disorders         
Adverse drug reaction  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Asthenia  1  0/6 (0.00%)  0/7 (0.00%)  1/1 (100.00%)  0/2 (0.00%) 
Fatigue  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Generalised oedema  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Pyrexia  1  2/6 (33.33%)  2/7 (28.57%)  0/1 (0.00%)  0/2 (0.00%) 
Infections and infestations         
Abscess limb  1  0/6 (0.00%)  0/7 (0.00%)  1/1 (100.00%)  0/2 (0.00%) 
Acute sinusitis  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Candidiasis  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Cellulitis  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Device related infection  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Ear infection  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Fungal skin infection  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Gastroenteritis viral  1  1/6 (16.67%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Gastrointestinal viral infection  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Hordeolum  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Influenza  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Nasopharyngitis  1  2/6 (33.33%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Otitis media  1  0/6 (0.00%)  2/7 (28.57%)  0/1 (0.00%)  0/2 (0.00%) 
Otitis media acute  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Pneumococcal infection  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Pneumonia  1  2/6 (33.33%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Rash pustular  1  0/6 (0.00%)  0/7 (0.00%)  1/1 (100.00%)  0/2 (0.00%) 
Respiratory tract infection bacterial  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Respiratory tract infection viral  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Sinusitis  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Streptococcal infection  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Subcutaneous abscess  1  0/6 (0.00%)  0/7 (0.00%)  1/1 (100.00%)  0/2 (0.00%) 
Tonsillitis  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Tooth abscess  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Upper respiratory tract infection  1  2/6 (33.33%)  3/7 (42.86%)  0/1 (0.00%)  0/2 (0.00%) 
Urinary tract infection  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Varicella  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Viral infection  1  2/6 (33.33%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Injury, poisoning and procedural complications         
Arthropod bite  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Contusion  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Epicondylitis  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Excoriation  1  0/6 (0.00%)  0/7 (0.00%)  1/1 (100.00%)  0/2 (0.00%) 
Fall  1  1/6 (16.67%)  1/7 (14.29%)  1/1 (100.00%)  1/2 (50.00%) 
Muscle strain  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Vaccination complication  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Investigations         
Aspiration tracheal abnormal  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Blood calcium increased  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Breath sounds abnormal  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Heart rate irregular  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Prostatic specific antigen increased  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Protein urine present  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
White blood cells urine positive  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Metabolism and nutrition disorders         
Dehydration  1  2/6 (33.33%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Gout  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Hypocalcaemia  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Hypoglycaemia  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Hypokalaemia  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Hypomagnesaemia  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Musculoskeletal and connective tissue disorders         
Back pain  1  1/6 (16.67%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Muscle spasms  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Myalgia  1  0/6 (0.00%)  2/7 (28.57%)  0/1 (0.00%)  0/2 (0.00%) 
Pain in extremity  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Nervous system disorders         
Dizziness  1  0/6 (0.00%)  0/7 (0.00%)  1/1 (100.00%)  0/2 (0.00%) 
Headache  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Hypotonia  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Presyncope  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Restless legs syndrome  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Somnolence  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Tremor  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Renal and urinary disorders         
Pollakiuria  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Asthma  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Atelectasis  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Cough  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Dyspnoea  1  1/6 (16.67%)  1/7 (14.29%)  0/1 (0.00%)  1/2 (50.00%) 
Increased bronchial secretion  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Oropharyngeal pain  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Vasomotor rhinitis  1  0/6 (0.00%)  0/7 (0.00%)  1/1 (100.00%)  0/2 (0.00%) 
Skin and subcutaneous tissue disorders         
Dermatitis diaper  1  1/6 (16.67%)  2/7 (28.57%)  0/1 (0.00%)  0/2 (0.00%) 
Eczema  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Erythema  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Rash  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Rash erythematous  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Rash macular  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Urticaria  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Vascular disorders         
Blood pressure fluctuation  1  0/6 (0.00%)  1/7 (14.29%)  0/1 (0.00%)  0/2 (0.00%) 
Hypotension  1  1/6 (16.67%)  0/7 (0.00%)  0/1 (0.00%)  0/2 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.0
This small exploratory study lacked a parallel control arm at the standard dose for a longer period; decline in respiratory or motor function prior to study was not collected systematically, thus change from baseline observations are inconclusive.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
In multi-site studies, PI will delay submission of a publication until the earlier of; (i) publication of the multi-center data by Genzyme (ii)12-24 months after study completion or termination of the Study at all sites, or (iii) Genzyme notification that a publication will not occur. PI will send Genzyme a draft 60 days before publication. Genzyme can defer publication 90-120 days upon notifying PI that it will file a patent application on inventions contained in the draft.
Results Point of Contact
Name/Title: Genzyme Medical Information
Organization: Genzyme Corporation
Phone: 800-745-4447
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00483379     History of Changes
Other Study ID Numbers: AGLU03306
First Submitted: June 6, 2007
First Posted: June 7, 2007
Results First Submitted: February 7, 2011
Results First Posted: April 22, 2011
Last Update Posted: March 7, 2014