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Study of rhASB in Patients With Mucopolysaccharidosis VI

This study has been completed.
Information provided by:
BioMarin Pharmaceutical Identifier:
First received: February 24, 2005
Last updated: January 26, 2010
Last verified: January 2010
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.

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

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:

Further study details as provided by BioMarin Pharmaceutical:

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

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

Enrollment: 39
Study Start Date: February 2004
Study Completion Date: October 2006
Primary Completion Date: October 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
N-acetylgalactosamine 4-sulfatase
Drug: N-acetylgalactosamine 4-sulfatase
Other Name: rhASB
Placebo/rhASB Drug: Placebo/rhASB


Ages Eligible for Study:   7 Years and older   (Child, Adult, Senior)
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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00104234

United States, California
BioMarin Pharmaceutical Inc.
Novato, California, United States, 94949
Sponsors and Collaborators
BioMarin Pharmaceutical
  More Information Identifier: NCT00104234     History of Changes
Other Study ID Numbers: ASB-03-06
Study First Received: February 24, 2005
Results First Received: February 4, 2009
Last Updated: January 26, 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 processed this record on September 20, 2017