COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC:

Get the latest research information from NIH: Menu

Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) 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. Identifier: NCT00067470
Recruitment Status : Completed
First Posted : August 25, 2003
Results First Posted : August 17, 2009
Last Update Posted : December 24, 2009
Information provided by:
BioMarin Pharmaceutical

Brief Summary:
The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk test at Week 24 compared with baseline.

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis VI Drug: Placebo Drug: N-acetylgalactosamine 4-sulfatase Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 39 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Treatment
Study Start Date : September 2003
Study Completion Date : March 2004

Arm Intervention/treatment
Placebo Comparator: Placebo Drug: Placebo
Active Comparator: rhASB Drug: N-acetylgalactosamine 4-sulfatase
Other Name: rhASB

Primary Outcome Measures :
  1. Change From Baseline in 12-minute Walk Test at 24 Weeks [ Time Frame: baseline and 24 weeks ]
    Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline

Secondary Outcome Measures :
  1. Change From Baseline in 3-minute Stair Climb at 24 Weeks [ Time Frame: baseline and 24 weeks ]
    Number of stairs climbed per minute in 3 minutes at 24 weeks minus number of stairs climbed per minute in 3 minutes at baseline

  2. Change From Baseline in Urinary GAG (uGAG) at 24 Weeks [ Time Frame: baseline and 24 weeks ]
    Glycosaminoglycan (GAG) level measured in urine

Information from the National Library of Medicine

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, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria

  • Patient consent
  • Patient must be seven years of age or older
  • Patient must have documented biochemical or genetic proof of MPS VI
  • The patient must be able to walk independently at least 5 meters and no more than 270 meters in the first 6 minutes, or no more then 400 meters total in 12 minutes, in the screening 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)
  • Patient refuses or is unable to complete all screening evaluations
  • 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
  • The patient has clinically significant spinal cord compression
  • The patient has known hypersensitivity to rhASB or to components of the active or placebo test solutions

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00067470

Layout table for location information
United States, California
BioMarin Pharmaceutical Inc.
Novato, California, United States, 94949
Sponsors and Collaborators
BioMarin Pharmaceutical
Additional Information:
Layout table for additonal information Identifier: NCT00067470    
Other Study ID Numbers: ASB-03-05
First Posted: August 25, 2003    Key Record Dates
Results First Posted: August 17, 2009
Last Update Posted: December 24, 2009
Last Verified: December 2009
Additional relevant MeSH terms:
Layout table for MeSH terms
Mucopolysaccharidosis VI
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases