Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

Mucopolysaccharidosis VI Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00067470
• Other study ID #: ASB-03-05
• Status: Completed
• Phase: Phase 3
• First received: August 20, 2003
• Last updated: December 18, 2009
• Start date: September 2003
• Est. completion date: March 2004

Study information

• Verified date: December 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 to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk test at Week 24 compared with baseline.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 39
• Est. completion date: March 2004
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 7 Years and older

Eligibility

Inclusion Criteria

• Patient consent

• Patient must be seven years of age or older

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

• The patient must be able to walk independently at least 5 meters and no more than 270 meters in the first 6 minutes, or no more then 400 meters total in 12 minutes, in the screening 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)

• Patient refuses or is unable to complete all screening evaluations

• 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

• The patient has clinically significant spinal cord compression

• The patient has known hypersensitivity to rhASB or to components of the active or placebo test solutions

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:
• Placebo

• 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	Change From Baseline in 12-minute Walk Test at 24 Weeks		baseline and 24 weeks	No
Secondary	Change From Baseline in 3-minute Stair Climb at 24 Weeks		baseline and 24 weeks	No
Secondary	Change From Baseline in Urinary GAG (uGAG) at 24 Weeks		baseline and 24 weeks	No

External Links

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