Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Mucopolysaccharidosis VI Clinical Trial

Official title:

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00048711
• Other study ID #: ASB-01-04
• Status: Completed
• Phase: Phase 2
• First received: November 6, 2002
• Last updated: August 24, 2009
• Start date: March 2002
• Est. completion date: June 2006

Study information

• Verified date: August 2009
• 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 evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (MPS VI)

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Est. completion date: June 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 5 Years and older

Eligibility

Inclusion Criteria:

• Patient consent

• Patient must be five years of age or older

• Patient must have documented biochemical or genetic proof of MPS VI

• Patient must walk at least 1 meter, but less than 250 meters, in the first 6 minutes of the baseline 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).

• 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

• Patient has a 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: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, 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	12-minute walk test		weeks 6, 12, 24, 48, 96 and 144	No
Primary	urinary glycoaminoglycan(uGAG)levels		weeks 1, 4, 6, 8, 12	No

External Links

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