A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

Completed

Conditions studied: MPS IIIB (Sanfilippo Syndrome)

In brief

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.

Key facts

Study ID

NCT02293382

Run by

Alexion Pharmaceuticals, Inc.

People needed

30

Starts

2013-07-01

Expected to finish

2015-01-01

Last updated by the study team

2017-02-03

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• The deceased patient was diagnosed with MPS IIIB as determined by either of the following:
• Documented deficiency of NAGLU enzyme activity.
• Documented functionally-relevant mutations in both alleles of the NAGLU gene.
• Patient's parent or legal guardian provides informed consent (unless the requirement for informed consent is waived by local regulations).
• The availability of pre-defined information in the patient's clinical chart:

You may not qualify if…

• There are no exclusion criteria for this study.

Where it is running

• Study site — Pittsburgh, Pennsylvania, United States
• Study site — Porte Alegre, Brazil
• Study site — Amsterdam, Netherlands
• Study site — Barcelona, Spain
• Study site — Birmingham, United Kingdom

Full record on ClinicalTrials.gov

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