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

Phase 1

Completed

• Conditions: Mucopolysaccharidosis VI

• Registration Number: NCT00048620
• Lead Sponsor: BioMarin Pharmaceutical

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2000-09
• Study First Submitted: 2002-11-04
• Study First Submitted QC: 2002-11-05
• Study First Posted: 2002-11-06
• Study Completion: 2005-11
• Status Verified: 2006-11
• Last Update Submitted: 2006-11-02
• Last Update Posted: 2006-11-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: Not specified

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)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
Lysosomal storage disease

Trial Locations

Locations (1)

BioMarin Pharmaceutical Inc.; 🇺🇸 Novato, California, United States