Back to resultsStudy of rhASB in Patients With Mucopolysaccharidosis VI
Primary Purpose
Mucopolysaccharidosis VI
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
N-acetylgalactosamine 4-sulfatase
Placebo/rhASB
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis VI
Eligibility Criteria
Inclusion Criteria: Patient consent Patient must have successfully completed study ASB-03-05, including having received at least 20 of 24 scheduled weekly infusions and having missed no more then two consecutive infusions If female of childbearing potential, patient must have a negative pregnancy test Exclusion Criteria: Pregnant or lactating patient Patient has received an investigational drug within 30 days prior to study enrollment Patient is unwilling or unable to travel to the primary site for periodic assessments
Sites / Locations
- BioMarin Pharmaceutical Inc.
Arms of the Study
Arm 1
Arm 2
Arm Type
Other
Other
Arm Label
rhASB/rhASB
Placebo/rhASB
Arm Description
N-acetylgalactosamine 4-sulfatase
Outcomes
Primary Outcome Measures
12-Minute Walk Test
Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.
Secondary Outcome Measures
3-Minute Stair Climb
Mean change in number of stairs climbed per minute in 3 minutes. Mean change is the mean difference between the 3-Minute Stair Climb at 96 weeks and that measured before first ever treatment with rhASB. Mean change is calculated for Week 96 - Baseline for the rhASB/rhASB group and for Week 96 - Week 24 for the placebo/rhASB group.
Change in Urinary Glycosaminoglycans (GAG) Level
Mean change in urinary GAG level for the first 72 weeks of rhASB treatment. For the rhASB/rhASB group, mean change was calculated for Week 72 -Baseline. For the placebo/rhASB, mean change was calculated for Week 96 - Week 24.
Full Information
NCT ID
NCT00104234
First Posted
February 24, 2005
Last Updated
January 26, 2010
Sponsor
BioMarin Pharmaceutical
1. Study Identification
Unique Protocol Identification Number
NCT00104234
Brief Title
Study of rhASB in Patients With Mucopolysaccharidosis VI
Official Title
A Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis VI
Study Type
Interventional
2. Study Status
Record Verification Date
January 2010
Overall Recruitment Status
Completed
Study Start Date
February 2004 (undefined)
Primary Completion Date
October 2006 (Actual)
Study Completion Date
October 2006 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
BioMarin Pharmaceutical
4. Oversight
5. Study Description
Brief Summary
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis VI
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
39 (Actual)
8. Arms, Groups, and Interventions
Arm Title
rhASB/rhASB
Arm Type
Other
Arm Description
N-acetylgalactosamine 4-sulfatase
Arm Title
Placebo/rhASB
Arm Type
Other
Intervention Type
Drug
Intervention Name(s)
N-acetylgalactosamine 4-sulfatase
Other Intervention Name(s)
rhASB
Intervention Type
Drug
Intervention Name(s)
Placebo/rhASB
Primary Outcome Measure Information:
Title
12-Minute Walk Test
Description
Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.
Time Frame
Baseline of ASB-03-05 through week 96 of ASB-03-06
Secondary Outcome Measure Information:
Title
3-Minute Stair Climb
Description
Mean change in number of stairs climbed per minute in 3 minutes. Mean change is the mean difference between the 3-Minute Stair Climb at 96 weeks and that measured before first ever treatment with rhASB. Mean change is calculated for Week 96 - Baseline for the rhASB/rhASB group and for Week 96 - Week 24 for the placebo/rhASB group.
Time Frame
Baseline ASB-03-05 through week 96 of ASB-03-06.
Title
Change in Urinary Glycosaminoglycans (GAG) Level
Description
Mean change in urinary GAG level for the first 72 weeks of rhASB treatment. For the rhASB/rhASB group, mean change was calculated for Week 72 -Baseline. For the placebo/rhASB, mean change was calculated for Week 96 - Week 24.
Time Frame
72 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patient consent
Patient must have successfully completed study ASB-03-05, including having received at least 20 of 24 scheduled weekly infusions and having missed no more then two consecutive infusions
If female of childbearing potential, patient must have a negative pregnancy test
Exclusion Criteria:
Pregnant or lactating patient
Patient has received an investigational drug within 30 days prior to study enrollment
Patient is unwilling or unable to travel to the primary site for periodic assessments
Facility Information:
Facility Name
BioMarin Pharmaceutical Inc.
City
Novato
State/Province
California
ZIP/Postal Code
94949
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
33874971
Citation
Tandon PK, Kakkis ED. The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases. Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5.
Results Reference
derived
Learn more about this trial
Study of rhASB in Patients With Mucopolysaccharidosis VI
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