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A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB | Clinical Trials | Clareo Health

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COMPLETED

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

NCT02293382•Alexion Pharmaceuticals, Inc.

View on ClinicalTrials.gov

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.

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

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:

Locations (5)

Pittsburgh, Pennsylvania, United States

Porte Alegre, Brazil

Amsterdam, Netherlands

Barcelona, Spain

Birmingham, United Kingdom

Key Dates

Start Date

July 1, 2013

Primary Completion Date

January 1, 2015

Completion Date

January 1, 2015

Last Updated

February 3, 2017

Enrollment

ACTUAL participants

Conditions

MPS IIIB (Sanfilippo Syndrome)