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

Primary Purpose

Mucopolysaccharidosis VI

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
N-acetylgalactosamine 4-sulfatase
Sponsored by
BioMarin Pharmaceutical
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis VI

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • BioMarin Pharmaceutical Inc.

Outcomes

Primary Outcome Measures

12-minute walk test
urinary glycoaminoglycan(uGAG)levels

Secondary Outcome Measures

Full Information

First Posted
November 6, 2002
Last Updated
August 24, 2009
Sponsor
BioMarin Pharmaceutical
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1. Study Identification

Unique Protocol Identification Number
NCT00048711
Brief Title
Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
Official Title
Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
Study Type
Interventional

2. Study Status

Record Verification Date
August 2009
Overall Recruitment Status
Completed
Study Start Date
March 2002 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
June 2006 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
BioMarin Pharmaceutical

4. Oversight

5. Study Description

Brief 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)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis VI

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
N-acetylgalactosamine 4-sulfatase
Primary Outcome Measure Information:
Title
12-minute walk test
Time Frame
weeks 6, 12, 24, 48, 96 and 144
Title
urinary glycoaminoglycan(uGAG)levels
Time Frame
weeks 1, 4, 6, 8, 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stuart J Sweidler, MD, Ph.D.
Organizational Affiliation
BioMarin Pharmaceutical
Official's Role
Study Director
Facility Information:
Facility Name
BioMarin Pharmaceutical Inc.
City
Novato
State/Province
California
ZIP/Postal Code
94949
Country
United States

12. IPD Sharing Statement

Links:
URL
http://www.bmrn.com
Description
Related Info

Learn more about this trial

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

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