Mucopolysaccharidosis VI Clinical Trial
Official title:
A Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis VI
Verified date | January 2010 |
Source | BioMarin Pharmaceutical |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Food and Drug Administration |
Study type | Interventional |
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
Status | Completed |
Enrollment | 39 |
Est. completion date | October 2006 |
Est. primary completion date | October 2006 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 7 Years and older |
Eligibility |
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 |
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | BioMarin Pharmaceutical Inc. | Novato | California |
Lead Sponsor | Collaborator |
---|---|
BioMarin Pharmaceutical |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 12-Minute Walk Test | Baseline of ASB-03-05 through week 96 of ASB-03-06 | No | |
Secondary | 3-Minute Stair Climb | Baseline ASB-03-05 through week 96 of ASB-03-06. | No | |
Secondary | Change in Urinary Glycosaminoglycans (GAG) Level | 72 weeks | No |
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