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

• Status: Completed
• Trial end date: 2005-11-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1
• 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 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)