Status:
COMPLETED
Longitudinal Studies of Brain Structure and Function in MPS Disorders
Lead Sponsor:
University of Minnesota
Collaborating Sponsors:
Rare Diseases Clinical Research Network
National Center for Advancing Translational Sciences (NCATS)
Conditions:
Mucopolysaccharidosis Type I
Mucopolysaccharidosis Type II
Eligibility:
All Genders
6+ years
Brief Summary
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 patient...
Detailed Description
The mucopolysaccharidoses (MPS diseases) are lysosomal disorders (inborn errors of metabolism) that progressively affect most organ systems in the body, usually beginning in childhood. Recent treatmen...
Eligibility Criteria
Inclusion
- Any MPS I, II, IV, VI or VII child or adult aged 6 years of age or older
Exclusion
- 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
Key Trial Info
Start Date :
September 1 2009
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
August 31 2019
Estimated Enrollment :
100 Patients enrolled
Trial Details
Trial ID
NCT01870375
Start Date
September 1 2009
End Date
August 31 2019
Last Update
September 6 2019
Active Locations (4)
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1
Oakland Children's Hospital
Oakland, California, United States, 94609
2
University of Minnesota
Minneapolis, Minnesota, United States, 55455
3
New York University
New York, New York, United States, 10016
4
Hospital for Sick Children
Toronto, Ontario, Canada, M5G1X8