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

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: Evaluation of Blood Brain Barrier Integrity and Relationship to Structural Brain Abnormalities in MPS IIIB Patients Using Cerebrospinal Fluid/Serum Albumin Index (CSF-AI)

Brief 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).

Detailed Summary:
Sponsor: Alexion Pharmaceuticals

Current Primary Outcome:

  • Blood Brain Barrier integrity in MPS IIIB subjects [ Time Frame: Day 0 ]
    Blood Brain Barrier integrity in MPS IIIB subjects by estimating the CSF-AI.
  • Blood Brain Barrier transfer coefficient [ Time Frame: Day 0 ]
    The Blood Brain Barrier transfer coefficient will be measured by DCE-MRI in MPS IIIB subjects.


Original Primary Outcome: Same as current

Current Secondary Outcome: Structural brain abnormalities in MPS IIIB [ Time Frame: Day 0 ]

Structural brain abnormalities in MPS IIIB using imaging and biomarkers related to underlying disease biology of MPS IIIB subjects


Original Secondary Outcome: Same as current

Information By: Alexion Pharmaceuticals

Dates:
Date Received: March 12, 2014
Date Started: December 2013
Date Completion:
Last Updated: June 23, 2016
Last Verified: June 2016