Overview

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)