Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging

Study Identifier:
NGLU-CL01
ClinicalTrials.gov Identifier:
NCT02090179
EudraCT Identifier:
2013-001938-18
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Unmapped
Study Drug
    Date
    Dec 2013 - May 2014
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Female & Male
    Age: 5+ years
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • Subject and/or subject's parent or legal guardian provides informed consent
    • Subject is ≥5 years of age.
    • Subject has a definitive diagnosis of MPS IIIB, as determined by either a documented deficiency in alpha-N-acetylglucosaminidase (NAGLU) enzyme activity or documented functionally-relevant mutations in both alleles of the NAGLU gene.
    Exclusion Criteria
    • The subject has any internal or non-removable external metal items that may present a safety risk (for MRI), or any other medical condition or circumstance in which an MRI is contraindicated.
    • The subject has a known or suspected hypersensitivity to anaesthesia, a bleeding disorder, or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated.
    • Previous allergic reaction to gadolinium-based MRI contrast media.
    Sex
    Female & Male
    Age
    5+ years

    Study Details

    Medical Condition
    • Unmapped
    Study Drug
      Date
      Dec 2013 - May 2014
      Phase 1
      Phase 2
      Phase 3
      Phase 4
      N/A
      Patient Requirements
      Sex: Female & Male
      Age: 5+ years years
      Requirements Information
      Inclusion and Exclusion Criteria
      Inclusion Criteria
      • Subject and/or subject's parent or legal guardian provides informed consent
      • Subject is ≥5 years of age.
      • Subject has a definitive diagnosis of MPS IIIB, as determined by either a documented deficiency in alpha-N-acetylglucosaminidase (NAGLU) enzyme activity or documented functionally-relevant mutations in both alleles of the NAGLU gene.
      Exclusion Criteria
      • The subject has any internal or non-removable external metal items that may present a safety risk (for MRI), or any other medical condition or circumstance in which an MRI is contraindicated.
      • The subject has a known or suspected hypersensitivity to anaesthesia, a bleeding disorder, or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated.
      • Previous allergic reaction to gadolinium-based MRI contrast media.

      Protocol Summary

      The purpose of the study is to characterize structural abnormalities in the brain and the integrity of the blood brain barrier in patients with mucopolysaccharidosis type IIIB (MPS IIIB).

      Trial Locations

      Location
      Status
      Location
      Birmingham, United Kingdom
      Status
      N/A