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

Mucopolysaccharidosis VI Clinical Trial

Official title:

Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00048620
• Other study ID #: ASB-00-01
• Status: Completed
• Phase: Phase 1
• First received: November 4, 2002
• Last updated: November 2, 2006
• Start date: September 2000
• Est. completion date: November 2005

Study information

• Verified date: November 2006
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Est. completion date: November 2005
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis VI

Intervention

Drug:
• N-acetylgalactosamine 4-sulfatase

Locations

Country	Name	City	State
United States	BioMarin Pharmaceutical Inc.	Novato	California

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Lysosomal storage disease

External Links

•   Link