Study of rhASB in Patients With Mucopolysaccharidosis 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: NCT00104234
Recruitment Status : Completed
First Posted : February 25, 2005
Results First Posted : March 6, 2009
Last Update Posted : February 2, 2010
Information provided by:
BioMarin Pharmaceutical

Brief Summary:
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 39 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis VI
Study Start Date : February 2004
Actual Primary Completion Date : October 2006
Actual Study Completion Date : October 2006

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

Placebo/rhASB Drug: Placebo/rhASB

Primary Outcome Measures :
  1. 12-Minute Walk Test [ Time Frame: Baseline of ASB-03-05 through week 96 of ASB-03-06 ]

Secondary Outcome Measures :
  1. 3-Minute Stair Climb [ Time Frame: Baseline ASB-03-05 through week 96 of ASB-03-06. ]
  2. Change in Urinary Glycosaminoglycans (GAG) Level [ Time Frame: 72 weeks ]

Information from the National Library of Medicine

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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 have successfully completed study ASB-03-05, including having received at least 20 of 24 scheduled weekly infusions and having missed no more then two consecutive infusions
  • If female of childbearing potential, patient must have a negative pregnancy test

Exclusion Criteria:

  • Pregnant or lactating patient
  • Patient has received an investigational drug within 30 days prior to study enrollment
  • Patient is unwilling or unable to travel to the primary site for periodic assessments

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): NCT00104234

United States, California
BioMarin Pharmaceutical Inc.
Novato, California, United States, 94949
Sponsors and Collaborators
BioMarin Pharmaceutical Identifier: NCT00104234     History of Changes
Other Study ID Numbers: ASB-03-06
First Posted: February 25, 2005    Key Record Dates
Results First Posted: March 6, 2009
Last Update Posted: February 2, 2010
Last Verified: January 2010

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