Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
Phase 2
Completed
- Conditions
- Mucopolysaccharidosis VI
- Registration Number
- NCT00048711
- Lead Sponsor
- BioMarin Pharmaceutical
- Brief Summary
The purpose of the study is evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (MPS VI)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria
- Patient consent
- Patient must be five years of age or older
- Patient must have documented biochemical or genetic proof of MPS VI
- Patient must walk at least 1 meter, but less than 250 meters, in the first 6 minutes of the baseline 12-minute walk test
- If female of childbearing potential, patient must have a negative pregnancy test
Exclusion Criteria
- Patient is under consideration for or has undergone a successful bone marrow transplant (BMT).
- Pregnant or lactating patient
- Patient has received an investigational drug within 30 days prior to study enrollment
- Patient has been previously treated with rhASB
- Patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly confound study results or decrease study compliance
- Patient has a known hypersensitivity to rhASB or to components of the study drug
- History of cancer (except low grade and fully resolved skin malignancy)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method 12-minute walk test weeks 6, 12, 24, 48, 96 and 144 urinary glycoaminoglycan(uGAG)levels weeks 1, 4, 6, 8, 12
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
BioMarin Pharmaceutical Inc.
🇺🇸Novato, California, United States