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

This study has been completed.
Sponsor:
Information provided by:
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT00048620
First received: November 4, 2002
Last updated: November 2, 2006
Last verified: November 2006
  Purpose

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 Intervention Phase
Mucopolysaccharidosis VI
Drug: N-acetylgalactosamine 4-sulfatase
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double-Blind
Primary Purpose: Treatment
Official Title: Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

Resource links provided by NLM:


Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • Lysosomal storage disease

Study Start Date: September 2000
Estimated Study Completion Date: November 2005
  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00048620

Locations
United States, California
BioMarin Pharmaceutical Inc.
Novato, California, United States, 94949
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
Study Director: Stuart J Swiedler, MD, Ph.D. BioMarin Pharmaceutical
  More Information

Additional Information:
No publications provided

ClinicalTrials.gov Identifier: NCT00048620     History of Changes
Other Study ID Numbers: ASB-00-01
Study First Received: November 4, 2002
Last Updated: November 2, 2006
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis VI
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on August 28, 2014