Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
NCT ID: NCT01733615
Last Updated: 2014-09-04
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
3 participants
OBSERVATIONAL
2012-06-30
2013-02-28
Brief Summary
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Detailed Description
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Conditions
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Study Design
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CASE_ONLY
PROSPECTIVE
Study Groups
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Mucopolysaccharidosis IVA
Patients with the condition.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
Exclusion Criteria
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Emory University
OTHER
Responsible Party
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Madhuri Hegde
Associate Professor
Principal Investigators
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Madhuri Hegde, PhD, FACMG
Role: PRINCIPAL_INVESTIGATOR
Emory University
Locations
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Emory University, Dept of Human Genetics
Decatur, Georgia, United States
Countries
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References
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Tiede S, Storch S, Lubke T, Henrissat B, Bargal R, Raas-Rothschild A, Braulke T. Mucolipidosis II is caused by mutations in GNPTA encoding the alpha/beta GlcNAc-1-phosphotransferase. Nat Med. 2005 Oct;11(10):1109-12. doi: 10.1038/nm1305. Epub 2005 Oct 2.
Walkley SU, Thrall MA, Haskins ME, Mitchell TW, Wenger DA, Brown DE, Dial S, Seim H. Abnormal neuronal metabolism and storage in mucopolysaccharidosis type VI (Maroteaux-Lamy) disease. Neuropathol Appl Neurobiol. 2005 Oct;31(5):536-44. doi: 10.1111/j.1365-2990.2005.00675.x.
Other Identifiers
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IRB00058450
Identifier Type: -
Identifier Source: org_study_id
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