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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 3-Minute Stair Climb [ Time Frame: Baseline ASB-03-05 through week 96 of ASB-03-06. ] [ Designated as safety issue: No ]
  • Change in Urinary Glycosaminoglycans (GAG) Level [ Time Frame: 72 weeks ] [ Designated as safety issue: No ]

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
Genders Eligible for Study:   Both
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
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

No publications provided 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
Health Authority: United States: Food and Drug Administration

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 April 17, 2014