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Study of rhASB in Patients With Mucopolysaccharidosis VI

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ClinicalTrials.gov Identifier: NCT00104234
Recruitment Status : Completed
First Posted : February 25, 2005
Results First Posted : March 6, 2009
Last Update Posted : February 2, 2010
Sponsor:
Information provided by:
BioMarin Pharmaceutical

Study Description
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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 Design
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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

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Arms and Interventions
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Arm Intervention/treatment
rhASB/rhASB
N-acetylgalactosamine 4-sulfatase
 Drug: N-acetylgalactosamine 4-sulfatase
Other Name: rhASB
Placebo/rhASB Drug: Placebo/rhASB


Outcome Measures
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Primary Outcome Measures :
  1. 12-Minute Walk Test [ Time Frame: Baseline of ASB-03-05 through week 96 of ASB-03-06 ]
    Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.


Secondary Outcome Measures :
  1. 3-Minute Stair Climb [ Time Frame: Baseline ASB-03-05 through week 96 of ASB-03-06. ]
    Mean change in number of stairs climbed per minute in 3 minutes. Mean change is the mean difference between the 3-Minute Stair Climb at 96 weeks and that measured before first ever treatment with rhASB. Mean change is calculated for Week 96 - Baseline for the rhASB/rhASB group and for Week 96 - Week 24 for the placebo/rhASB group.

  2. Change in Urinary Glycosaminoglycans (GAG) Level [ Time Frame: 72 weeks ]
    Mean change in urinary GAG level for the first 72 weeks of rhASB treatment. For the rhASB/rhASB group, mean change was calculated for Week 72 -Baseline. For the placebo/rhASB, mean change was calculated for Week 96 - Week 24.


Eligibility Criteria
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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
Criteria

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
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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 ClinicalTrials.gov identifier (NCT number): NCT00104234


Locations
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United States, California
BioMarin Pharmaceutical Inc.
Novato, California, United States, 94949
Sponsors and Collaborators
BioMarin Pharmaceutical
More Information
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ClinicalTrials.gov Identifier: NCT00104234    
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:
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Mucopolysaccharidoses
Mucopolysaccharidosis VI
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
 Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases