Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

BioMarin Pharmaceutical

Status and phase

Completed

Phase 1

Conditions

Mucopolysaccharidosis VI

Treatments

Drug: N-acetylgalactosamine 4-sulfatase

Study type

Interventional

Funder types

Industry

Identifiers

NCT00048620

ASB-00-01

Details and patient eligibility

About

The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in patients diagnosed with MPS VI.

Sex

All

Volunteers

No Healthy Volunteers

Inclusion criteria

Patient consent
Patient must be five years of age or older
Patient must have documented diagnosis of MPS VI, confirmed at screening by measurable clinical signs and symptoms of MPS VI
Leukocyte ASB enzyme activity level less than 20% of the normal range
Clinical evidence of significant MPS VI disease that provides adequate opportunity to achieve quantitative, short-term therapeutic benefit in three or more of the following parameters: endurance (as measured by a six-minute walk test), forced vital capacity (as measured by spirometry), joint range of motion, urinary glycosaminoglycans, and hepatomegaly.
Ability to perform all protocol tests
Ability to stand independently for six minutes
Sexually active subjects must agree to use an adequate form of contraception

Exclusion criteria

History of bone marrow transplantation
Pregnant or lactating patient
Use of an investigational drug or device within 30 days prior to study participation.
A medical condition, serious intercurrent illness, or other extenuating circumstances that may significantly decrease study compliance including prescribed follow-up
Known hypersensitivity to rhASB or to components of the study drug
History of cancer (except low grade and fully resolved skin malignancy)