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

Sponsor
BioMarin Pharmaceutical (Industry)
Overall Status
Completed
CT.gov ID
NCT00048711
Collaborator
(none)
1
Location
51
Duration (Months)

Study Details

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)

Condition or Disease Intervention/Treatment Phase
  • Drug: N-acetylgalactosamine 4-sulfatase
 Phase 2

Study Design

Study Type:
Interventional
Allocation:
Non-Randomized
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
Study Start Date :
Mar 1, 2002
Actual Study Completion Date :
Jun 1, 2006

Outcome Measures

Primary Outcome Measures

  1. 12-minute walk test [weeks 6, 12, 24, 48, 96 and 144]

  2. urinary glycoaminoglycan(uGAG)levels [weeks 1, 4, 6, 8, 12]

Eligibility Criteria

Criteria

Ages Eligible for Study:
5 Years 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 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)

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 Sweidler, MD, Ph.D., BioMarin Pharmaceutical

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

Publications

None provided.
Responsible Party:
, ,
ClinicalTrials.gov Identifier:
NCT00048711
Other Study ID Numbers:
  • ASB-01-04
First Posted:
Nov 8, 2002
Last Update Posted:
Aug 25, 2009
Last Verified:
Aug 1, 2009
Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis VI

Study Results

No Results Posted as of Aug 25, 2009