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

Primary Purpose

Mucopolysaccharidosis VI

Status
Completed
Phase
Phase 1
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

undefined - 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 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)

Sites / Locations

  • BioMarin Pharmaceutical Inc.

Outcomes

Primary Outcome Measures

Lysosomal storage disease

Secondary Outcome Measures

Full Information

First Posted
November 4, 2002
Last Updated
November 2, 2006
Sponsor
BioMarin Pharmaceutical
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1. Study Identification

Unique Protocol Identification Number
NCT00048620
Brief Title
Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
Official Title
Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
Study Type
Interventional

2. Study Status

Record Verification Date
November 2006
Overall Recruitment Status
Completed
Study Start Date
September 2000 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
November 2005 (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
BioMarin Pharmaceutical

4. Oversight

5. Study Description

Brief 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.

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 1
Interventional Study Model
Parallel Assignment
Masking
Double
Allocation
Randomized

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
N-acetylgalactosamine 4-sulfatase
Primary Outcome Measure Information:
Title
Lysosomal storage disease

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stuart J Swiedler, 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

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

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