A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
Study Identifier:
NGLU-NH01
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete
Study Details
Medical Condition
- Unmapped
Study Drug
Date
Jul 2013 - Jan 2015
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A
Requirements Information
Inclusion and Exclusion Criteria
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:
Exclusion Criteria
- There are no exclusion criteria for this study.
Sex
Female & Male
Age
N/A
Study Details
Medical Condition
- Unmapped
Study Drug
Date
Jul 2013 - Jan 2015
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A years
Requirements Information
Inclusion and Exclusion Criteria
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:
Exclusion Criteria
- There are no exclusion criteria for this study.
Protocol 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.
Trial Locations
Location
Status
Location
Pittsburgh, Pennsylvania, United States
Status
N/A
Location
Porte Alegre, Brazil
Status
N/A
Location
Amsterdam, Netherlands
Status
N/A
Location
Barcelona, Spain
Status
N/A
Location
Birmingham, United Kingdom
Status
N/A