Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

BioMarin Pharmaceutical

Status and phase

Completed

Phase 3

Conditions

Mucopolysaccharidosis VI

Treatments

Drug: Placebo

Drug: N-acetylgalactosamine 4-sulfatase

Study type

Interventional

Funder types

Industry

Identifiers

NCT00067470

ASB-03-05

Details and patient eligibility

About

The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk test at Week 24 compared with baseline.

Enrollment

39 patients

Sex

All

Ages

7+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

Patient consent
Patient must be seven years of age or older
Patient must have documented biochemical or genetic proof of MPS VI
The patient must be able to walk independently at least 5 meters and no more than 270 meters in the first 6 minutes, or no more then 400 meters total in 12 minutes, in the screening 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)
Patient refuses or is unable to complete all screening evaluations
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
The patient has clinically significant spinal cord compression
The patient has known hypersensitivity to rhASB or to components of the active or placebo test solutions