Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

• Status: Completed
• Trial end date: 2006-06-01
• Target enrollment: 0
• Participant gender: 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 2
• Accepts Healthy Volunteers?: No

Details

• 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)