A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
NCT ID: NCT00787995
Last Updated: 2014-12-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
353 participants
OBSERVATIONAL
2008-10-31
2014-07-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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CASE_ONLY
Study Groups
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MPS IVA
Subjects with mucopolysaccharidosis IVA (Morquio Syndrome)
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.
* Willing and able to comply with all study procedures.
Exclusion Criteria
* Previous hematopoietic stem cell transplant (HSCT).
* Concurrent disease or condition that would interfere with study participation or pose a safety concern.
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Celeste Decker, MD
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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Oakland, California, United States
Chicago, Illinois, United States
Córdoba, , Argentina
Porto Alegre, , Brazil
São Paulo, , Brazil
Montreal, Quebec, Canada
Lyon, , France
Paris, , France
Mainz, , Germany
Monza, , Italy
Amsterdam, , Netherlands
Taipei, , Taiwan
Birmingham, , United Kingdom
London, , United Kingdom
Manchester, , United Kingdom
Countries
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References
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Martell LA, Cunico RL, Ohh J, Fulkerson W, Furneaux R, Foehr ED. Validation of an LC-MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome. Bioanalysis. 2011 Aug;3(16):1855-66. doi: 10.4155/bio.11.172.
Related Links
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BioMarin Pharmaceutical Inc. website
Other Identifiers
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MOR-001
Identifier Type: -
Identifier Source: org_study_id