NCT00048711CompletedPhase 2
Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
Conditions
Drugs
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- BioMarin Pharmaceutical
- Locations
- 2
- Primary Endpoint
- 12-minute walk test
Study Overview
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)
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 5 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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)
Outcomes
Primary Outcomes
12-minute walk test
Time Frame: weeks 6, 12, 24, 48, 96 and 144
urinary glycoaminoglycan(uGAG)levels
Time Frame: weeks 1, 4, 6, 8, 12
Secondary Outcomes
No secondary outcomes reported
Investigators
Study Sites (2)
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