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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

5 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-12-31

Study Completion Date

2014-05-31

Brief Summary

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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).

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Detailed Description

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Conditions

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MPS IIIB (Sanfilippo B Syndrome)

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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MPS IIIB

Those with a definitive diagnosis of MPS IIIB (Sanfilippo B Syndrome).

No interventions assigned to this group

Eligibility Criteria

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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.

Minimum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No