Overview
Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
Status:
Completed
Completed
Trial end date:
2006-06-01
2006-06-01
Target enrollment:
0
0
Participant gender:
All
All
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)Phase:
Phase 2Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
BioMarin Pharmaceutical
Criteria
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)