Status:
COMPLETED
A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
MPS IIIB (Sanfilippo Syndrome)
Eligibility:
All Genders
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.
Eligibility Criteria
Inclusion
- 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:
Exclusion
- There are no exclusion criteria for this study.
Key Trial Info
Start Date :
July 1 2013
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
January 1 2015
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT02293382
Start Date
July 1 2013
End Date
January 1 2015
Last Update
February 3 2017
Active Locations (5)
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1
Pittsburgh, Pennsylvania, United States
2
Porte Alegre, Brazil
3
Amsterdam, Netherlands
4
Barcelona, Spain