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Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI

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ClinicalTrials.gov Identifier: NCT02437253
Recruitment Status : Completed
First Posted : May 7, 2015
Results First Posted : September 15, 2017
Last Update Posted : September 15, 2017
Sponsor:
Information provided by (Responsible Party):
Los Angeles Biomedical Research Institute

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Mucopolysaccharidosis Type I
Mucopolysaccharidosis Type II
Mucopolysaccharidosis Type VI
Interventions Drug: Adalimumab
Other: Placebo
Enrollment 2
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Adalimumab First, Then Placebo Placebo First, Then Adalimumab
Hide Arm/Group Description Participants first received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week. Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
Period Title: Overall Study
Started 1 1
Completed 1 1
Not Completed 0 0
Arm/Group Title Adalimumab First, Then Placebo Placebo First, Then Adalimumab Total
Hide Arm/Group Description Participants first received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Total of all reporting groups
Overall Number of Baseline Participants 1 1 2
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 1 participants 2 participants
<=18 years
1
 100.0%
1
 100.0%
2
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 1 participants 1 participants 2 participants
15
(15 to 15)
6
(6 to 6)
10.5
(6 to 15)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 1 participants 2 participants
Female
1
 100.0%
1
 100.0%
2
 100.0%
Male
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 1 participants 2 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
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
1
 100.0%
1
 100.0%
2
 100.0%
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%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 1 participants 1 participants 2 participants
1 1 2
1.Primary Outcome
Title Children's Health Questionnaire - Parent Form 50 Bodily Pain Standardized Score
Hide Description Children's Health Questionnaire - Parent Form 50 bodily pain (BP) standardized score for participants < 18 years of age. Range is from 0 to 100 (no units) and standardization if determined by comparison to healthy age matched children. Lower values mean increased pain. The difference of change in BP standardized score from day 0 to week 16 during adalimumab treatment minus change in BP standardized score from day 0 to week 16 during placebo treatment is reported.
Time Frame day 0 to week 16 of treatment with adalimumab versus placebo
Hide Outcome Measure Data
Hide Analysis Population Description
This was a cross-over study so within-individual changes are reported.
Arm/Group Title Adalimumab First, Then Placebo Placebo First, Then Adalimumab
Hide Arm/Group Description:
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks.
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
Overall Number of Participants Analyzed 1 1
Mean (Full Range)
Unit of Measure: units on a scale
1.4
(1.4 to 1.4)
1.0
(1.0 to 1.0)
2.Secondary Outcome
Title Children's Health Questionnaire - Parent Form 50 Physical Function (PF) Standardized Score
Hide Description Children's Health Questionnaire - Parent Form 50 physical function (PF) standardized score for participants < 18 years of age. Range is from 0 to 100 (no units) and standardization if determined by comparison to healthy age matched children. Lower values mean decreased physical function. The difference of change in PF standardized score from day 0 to week 16 during adalimumab treatment minus change in PF standardized score from day 0 to week 16 during placebo treatment is reported.
Time Frame Day 0 to week 16 of treatment with adalimumab versus placebo
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Adalimumab First, Then Placebo Placebo First, Then Adalimumab
Hide Arm/Group Description:
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks.
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
Overall Number of Participants Analyzed 1 1
Mean (Full Range)
Unit of Measure: units on a scale
0
(0 to 0)
0.4
(0.4 to 0.4)
3.Secondary Outcome
Title Pain Measured by the Visual Analog Scale (VAS) in the Pediatric Pain Questionnaire (PPQ)
Hide Description Percent of participants with a >10 mm improvement in either "how you feel now" or "worst pain you had this week" on the VA in the PPQ during adalimumab versus during placebo by either parental or subject report. Range is 0-100 mm; lower number means less pain.
Time Frame Day 0 to week 16 of treatment with adalimumab versus placebo
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Adalimumab First, Then Placebo Placebo First, Then Adalimumab
Hide Arm/Group Description:
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks.
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
Overall Number of Participants Analyzed 1 1
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
1
 100.0%
4.Secondary Outcome
Title Range of Motion - Bilateral Shoulder, Elbow, Hip, Knee
Hide Description Number of joints with a >5 degree more positive change during 16 weeks of adalimumab versus 16 weeks of placebo. A total of eight joints were measured.
Time Frame Day 0 to week 16 of treatment with adalimumab versus placebo
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Adalimumab First, Then Placebo Placebo First, Then Adalimumab
Hide Arm/Group Description:
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week.
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
Overall Number of Participants Analyzed 1 1
Measure Type: Number
Unit of Measure: joints
7 5
5.Secondary Outcome
Title Anti-ERT Antibodies
Hide Description Anti-laronidase antibodies for subjects with MPS I. Anti-idursulfase antibodies for subjects with MPS II.
Time Frame Day 0 to week 16 of treatment with adalimumab versus placebo
Hide Outcome Measure Data
Hide Analysis Population Description
No participants had anti-ERT antibodies
Arm/Group Title Adalimumab First, Then Placebo Placebo First, Then Adalimumab
Hide Arm/Group Description:
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week.
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
Overall Number of Participants Analyzed 1 1
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Adalimumab Placebo
Hide Arm/Group Description

20 mg subQ every other week (weight 15 to <30 kg) 40 mg subQ every other week (weight ≥30 kg).

Adalimumab: Subjects will be treated with adalimumab (20 mg [weight 15-<30 kg] or 40 mg [weight ≥30 kg] administered subcutaneously [subQ] every other week) or placebo for 16 weeks, then cross-over to the other group for 16 weeks.

Saline placebo injection

Placebo: Saline placebo

All-Cause Mortality
Adalimumab Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/2 (0.00%)      0/2 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Adalimumab Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/2 (0.00%)      0/2 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Adalimumab Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/2 (100.00%)      2/2 (100.00%)    
Blood and lymphatic system disorders     
Thrombocytopenia  [1]  1/2 (50.00%)  1 0/2 (0.00%)  0
Gastrointestinal disorders     
Diarrhea  [2]  1/2 (50.00%)  1 0/2 (0.00%)  0
Infections and infestations     
Upper Respiratory Infection   0/2 (0.00%)  0 2/2 (100.00%)  2
Sinusitis   0/2 (0.00%)  0 1/2 (50.00%)  1
Nervous system disorders     
Headache   0/2 (0.00%)  0 1/2 (50.00%)  1
Psychiatric disorders     
Mood lability   0/2 (0.00%)  0 1/2 (50.00%)  1
Skin and subcutaneous tissue disorders     
Injection site erythema   1/2 (50.00%)  1 0/2 (0.00%)  0
Indicates events were collected by systematic assessment
[1]
Resolved by 2 week recheck without intervention
[2]
Resolved after 2 days without intervention
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Name/Title: Dr. Lynda Polgreen (PI)
Organization: Los Angeles Biomedical Research Institute at Harbor-UCLA
Phone: 310-222-1961
Responsible Party: Los Angeles Biomedical Research Institute
ClinicalTrials.gov Identifier: NCT02437253     History of Changes
Other Study ID Numbers: 30535
First Submitted: May 5, 2015
First Posted: May 7, 2015
Results First Submitted: May 3, 2017
Results First Posted: September 15, 2017
Last Update Posted: September 15, 2017