A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

Study Identifier:
NGLU-NH01
ClinicalTrials.gov Identifier:
NCT02293382
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