Longitudinal Studies of Brain Structure and Function in MPS Disorders

Status: Completed
Location: See all (4) locations...
Study Type: Observational
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 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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: No
View:

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

Locations
United States
California
Oakland Children's Hospital
Oakland
Minnesota
University of Minnesota
Minneapolis
New York
New York University
New York
Other Locations
Canada
Hospital for Sick Children
Toronto
Time Frame
Start Date: September 2009
Completion Date: August 31, 2019
Participants
Target number of participants: 100
Treatments
MPS IH, MPS IHS, MPS IS
MPS IH (Hurler syndrome) patients; MPS IHS (Hurler-Scheie syndrome) patients; and MPS IS (Scheie syndrome) patients
MPS II
Hunter syndrome patients
MPS IV
Morquio syndrome patients who will be considered for enrollment in the study on an individual basis
MPS VI
Maroteaux-Lamy syndrome patients
MPS VII
Sly syndrome patients who will be considered for enrollment in the study on an individual basis
Sponsors
Collaborators: National Center for Advancing Translational Sciences (NCATS), Lysosomal Disease Network, National Institute of Neurological Disorders and Stroke (NINDS), Rare Diseases Clinical Research Network, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Leads: University of Minnesota

This content was sourced from clinicaltrials.gov