A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
NCT ID: NCT02293382
Last Updated: 2017-02-03
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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COMPLETED
30 participants
OBSERVATIONAL
2013-07-31
2015-01-31
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
RETROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
1. Documented deficiency of NAGLU enzyme activity.
2. Documented functionally-relevant mutations in both alleles of the NAGLU gene.
2. Patient's parent or legal guardian provides informed consent (unless the requirement for informed consent is waived by local regulations).
3. The availability of pre-defined information in the patient's clinical chart:
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Pittsburgh, Pennsylvania, United States
Porte Alegre, , Brazil
Amsterdam, , Netherlands
Barcelona, , Spain
Birmingham, , United Kingdom
Countries
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Other Identifiers
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NGLU-NH01
Identifier Type: -
Identifier Source: org_study_id
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