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

BioMarin Pharmaceutical

Status and phase

Completed

Phase 2

Conditions

Mucopolysaccharidosis VI

Treatments

Drug: N-acetylgalactosamine 4-sulfatase

Study type

Interventional

Funder types

Industry

Identifiers

NCT00048711

ASB-01-04

Details and patient eligibility

About

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)

Sex

All

Ages

5+ years old

Volunteers

No Healthy Volunteers

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)