Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
NCT ID: NCT00067470
Last Updated: 2009-12-24
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
39 participants
INTERVENTIONAL
2003-09-30
2004-03-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Placebo
Placebo
rhASB
N-acetylgalactosamine 4-sulfatase
Interventions
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Placebo
N-acetylgalactosamine 4-sulfatase
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Patient must be seven years of age or older
* Patient must have documented biochemical or genetic proof of MPS VI
* The patient must be able to walk independently at least 5 meters and no more than 270 meters in the first 6 minutes, or no more then 400 meters total in 12 minutes, in the screening 12-minute walk test
* If female of childbearing potential, patient must have a negative pregnancy test
Exclusion Criteria
* Patient refuses or is unable to complete all screening evaluations
* Pregnant or lactating patient
* Patient has received an investigational drug within 30 days prior to study enrollment
* Patient has been previously treated with rhASB
* Patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly confound study results or decrease study compliance
* The patient has clinically significant spinal cord compression
* The patient has known hypersensitivity to rhASB or to components of the active or placebo test solutions
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.
Related Links
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Related Info
Other Identifiers
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ASB-03-05
Identifier Type: -
Identifier Source: org_study_id