Study of rhASB in Patients With Mucopolysaccharidosis VI
NCT ID: NCT00104234
Last Updated: 2010-02-02
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
39 participants
INTERVENTIONAL
2004-02-29
2006-10-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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rhASB/rhASB
N-acetylgalactosamine 4-sulfatase
N-acetylgalactosamine 4-sulfatase
Placebo/rhASB
Placebo/rhASB
Interventions
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N-acetylgalactosamine 4-sulfatase
Placebo/rhASB
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
7 Years
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Locations
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BioMarin Pharmaceutical Inc.
Novato, California, United States
Countries
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References
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Tandon PK, Kakkis ED. The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases. Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5.
Other Identifiers
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ASB-03-06
Identifier Type: -
Identifier Source: org_study_id
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