Longitudinal Studies of Brain Structure and Function in MPS Disorders

Completed

Conditions studied: Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII

In brief

Neurobehavioral function and quality of life are compromised in many patients with mucopolysaccharidosis (MPS) disorders. The long-term goals of this research are to: 1) more accurately inform patients/parents regarding potential neurobehavioral outcomes; 2) develop sensitive measures of disease progression and central nervous system (CNS) treatment outcome; and 3) help clinical researchers develop direct treatments for specific brain structures/functions. The investigators hypothesize that specific and localized neuroimaging and neuropsychological findings and their relationship will be distinct for each MPS disorder. It is further hypothesized that without treatment, functions will decline and structure will change over time in a predictable fashion, and will be related to locus of abnormality and stage of disease.

Key facts

Study ID

NCT01870375

Run by

University of Minnesota

People needed

100

Starts

2009-09-01

Expected to finish

2019-08-31

Last updated by the study team

2019-09-06

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Any MPS I, II, IV, VI or VII child or adult aged 6 years of age or older

You may not qualify if…

• Exclusion Criteria for Neuroimaging:
• Participants with:
• Pacemakers
• Any indwelling electronic device including programmable shunts
• Orthodontic braces unless they are not made of metal
• Other implanted metal in the body other than titanium
• Unable to stay still during MRI because of low cognitive function, behavioral dysregulation, or young age, if the patient is not a clinical patient having sedation/anesthesia
• Pregnancy
• Exclusion Criteria for Neuropsychological and Neurobehavioral Testing
• Participants who:
• Are too functionally impaired for testing

Where it is running

• Oakland Children's Hospital — Oakland, California, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• New York University — New York, New York, United States
• Hospital for Sick Children — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

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