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A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

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Disease Information

Descriptive Information
Brief Title † A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
Official Title † A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
Brief Summary The objective is to perform a retrospective chart review to generate data to evaluate the clinical characteristics and course of disease progression of MPS IIIB.
Detailed Description
Study Phase N/A
Study Type † Observational
Study Design †
Primary Outcome Measure † Evaluation of disease presentation and the course of disease progression including the end stage of the disease in patients with MPS IIIB, assessed from medical history data obtained by retrospective chart review of deceased patients.
Secondary Outcome Measure †
Condition † MPS IIIB (Sanfilippo Syndrome)
Intervention †
Study Arms / Comparison Groups
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status †
Estimated Enrollment † 30
Start Date † July 2013
Completion Date January 2015
Primary Completion Date January 2015
Eligibility Criteria † Inclusion Criteria: 1. The deceased patient was diagnosed with MPS IIIB as determined by either of the following: 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: Exclusion Criteria: There are no exclusion criteria for this study.
Gender All
Ages N/A - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † Brazil
Administrative Information
NCT ID † NCT02293382
Organization ID NGLU-NH01
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Alexion Pharmaceuticals
Collaborators ††
Investigators † : ,
Information Provided By
Verification Date February 2017
First Received Date † November 3, 2014
Last Updated Date February 2, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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