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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00048711
Recruitment Status : Completed
First Posted : November 8, 2002
Last Update Posted : August 25, 2009
Sponsor:
Information provided by:
BioMarin Pharmaceutical

Tracking Information
First Submitted Date  ICMJE November 6, 2002
First Posted Date  ICMJE November 8, 2002
Last Update Posted Date August 25, 2009
Study Start Date  ICMJE March 2002
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE
 (submitted: August¬†24,¬†2009)
  • 12-minute walk test [ Time Frame: weeks 6, 12, 24, 48, 96 and 144 ]
  • urinary glycoaminoglycan(uGAG)levels [ Time Frame: weeks 1, 4, 6, 8, 12 ]
Original Primary Outcome Measures  ICMJE Not Provided
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
Official Title  ICMJE Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
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)
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Mucopolysaccharidosis VI
Intervention  ICMJE Drug: N-acetylgalactosamine 4-sulfatase
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Enrollment  ICMJE Not Provided
Original Enrollment  ICMJE Not Provided
Actual Study Completion Date  ICMJE June 2006
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

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)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00048711
Other Study ID Numbers  ICMJE ASB-01-04
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE BioMarin Pharmaceutical
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Stuart J Sweidler, MD, Ph.D. BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date August 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP