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

December 18, 2009 updated by: BioMarin Pharmaceutical
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

39

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Novato, California, United States, 94949
        • BioMarin Pharmaceutical Inc.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

7 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: Placebo
Active Comparator: rhASB
Drug: N-acetylgalactosamine 4-sulfatase
Other Names:
  • rhASB

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in 12-minute Walk Test at 24 Weeks
Time Frame: baseline and 24 weeks
Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
baseline and 24 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in 3-minute Stair Climb at 24 Weeks
Time Frame: baseline and 24 weeks
Number of stairs climbed per minute in 3 minutes at 24 weeks minus number of stairs climbed per minute in 3 minutes at baseline
baseline and 24 weeks
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks
Time Frame: baseline and 24 weeks
Glycosaminoglycan (GAG) level measured in urine
baseline and 24 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioMarin Pharmaceutical

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2003

Study Completion

March 1, 2004

Study Registration Dates

First Submitted

August 20, 2003

First Submitted That Met QC Criteria

August 22, 2003

First Posted (Estimate)

August 25, 2003

Study Record Updates

Last Update Posted (Estimate)

December 24, 2009

Last Update Submitted That Met QC Criteria

December 18, 2009

Last Verified

December 1, 2009

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ASB-03-05

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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