Evaluation of Blood Brain Barrier Integrity and Structura... | NCT02090179

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

NCT02090179

Sponsor

Alexion Pharmaceuticals, Inc.

Status

Completed

Last Update Posted

Jun 27, 2016Estimated

Enrollment

5Actual

Phase

Not provided

Conditions

MPS IIIB (Sanfilippo B Syndrome)

Interventions

Not provided

Countries

United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
2013-001938-18	EudraCT Number

Status Module

Record Verification Date

Jun 2016

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Dec 2013

Primary Completion Date

May 2014Actual

Completion Date

May 2014Actual

First Submitted Date

Mar 12, 2014

First Submission Date that Met QC Criteria

Mar 14, 2014

First Posted Date

Mar 18, 2014Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jun 23, 2016

Last Update Posted Date

Jun 27, 2016Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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 Description

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

5Actual

Label	Type	Description	Intervention Names
MPS IIIB		Those with a definitive diagnosis of MPS IIIB (Sanfilippo B Syndrome).

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Measure	Description	Time Frame
Structural brain abnormalities in MPS IIIB	Structural brain abnormalities in MPS IIIB using imaging and biomarkers related to underlying disease biology of MPS IIIB subjects	Day 0

Other Outcomes

Not provided

Eligibility Module

Eligibility 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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

5 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Up to six subjects with MPS IIIB. An attempt will be made to enroll equal numbers (three) of MPS IIIB subjects with a classic (severe) disease presentation, and MPS IIIB subjects with an attenuated phenotype.

Sampling Method

Non-Probability Sample

Condition Browse Module

MeSH Terms

ID	Term
D009084	Mucopolysaccharidosis III
D009083	Mucopolysaccharidoses
D008661	Metabolism, Inborn Errors
D008659	Metabolic Diseases
D030342	Genetic Diseases, Inborn

Ancestor Terms

ID	Term
D002239	Carbohydrate Metabolism, Inborn Errors
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D016464	Lysosomal Storage Diseases
D017520	Mucinoses

Browse Leaves

Not provided

Browse Branches

Not provided