Full Text View
Tabular View
No Study Results Posted
Related Studies
Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
This study has been completed.
Study NCT00048620   Information provided by BioMarin Pharmaceutical
First Received: November 4, 2002   Last Updated: November 2, 2006   History of Changes

November 4, 2002
November 2, 2006
September 2000
 
Lysosomal storage disease
Same as current
Complete list of historical versions of study NCT00048620 on ClinicalTrials.gov Archive Site
 
 
 
Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

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.

 
Phase I
Interventional
Treatment, Randomized, Double-Blind, Dose Comparison, Parallel Assignment, Safety/Efficacy Study
Mucopolysaccharidosis VI
Drug: N-acetylgalactosamine 4-sulfatase
 
 

*   Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
 
Completed
 
November 2005
 

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)
Both
 
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00048620
 
ASB-00-01
BioMarin Pharmaceutical
 
Study Director: Stuart J Swiedler, MD, Ph.D. BioMarin Pharmaceutical
BioMarin Pharmaceutical
November 2006

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