CompletedNot Applicable

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

United States, Brazil, Netherlands30 participantsStarted 2013-07

Plain-language 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.

Who can participate

SexALL

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Inclusion criteria

✓. 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:

What they're measuring

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.

Timeframe: N/A as this is a retrospective chart review of deceased patients

Trial details

NCT IDNCT02293382

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2015-01

View on ClinicalTrials.gov More MPS IIIB (Sanfilippo Syndrome) trials