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

Sponsor
BioMarin Pharmaceutical (Industry)
Overall Status
Completed
CT.gov ID
NCT00048620
Collaborator
(none)
1
Location
62
Duration (Months)

Study Details

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: N-acetylgalactosamine 4-sulfatase
 Phase 1

Study Design

Study Type:
Interventional
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Double
Primary Purpose:
Treatment
Official Title:
Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
Study Start Date :
Sep 1, 2000
Study Completion Date :
Nov 1, 2005

Outcome Measures

Primary Outcome Measures

  1. Lysosomal storage disease []

Eligibility Criteria

Criteria

Ages Eligible for Study:
N/A and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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)

Contacts and Locations

Locations

Site City State Country Postal Code
1 BioMarin Pharmaceutical Inc. Novato California United States 94949

Sponsors and Collaborators

  • BioMarin Pharmaceutical

Investigators

  • Study Director: Stuart J Swiedler, MD, Ph.D., BioMarin Pharmaceutical

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

Publications

None provided.
Responsible Party:
, ,
ClinicalTrials.gov Identifier:
NCT00048620
Other Study ID Numbers:
  • ASB-00-01
First Posted:
Nov 6, 2002
Last Update Posted:
Nov 3, 2006
Last Verified:
Nov 1, 2006
Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis VI

Study Results

No Results Posted as of Nov 3, 2006