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Longitudinal Studies of Brain Structure and Function in MPS Disorders

NCT01870375 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 100

Last updated 2019-09-06

No results posted yet for this study

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.

Conditions

  • Mucopolysaccharidosis Type I
  • Mucopolysaccharidosis Type II
  • Mucopolysaccharidosis Type VI
  • Mucopolysaccharidosis Type IV
  • Mucopolysaccharidosis Type VII

Sponsors & Collaborators

  • Rare Diseases Clinical Research Network

    collaborator NETWORK

  • National Center for Advancing Translational Sciences (NCATS)

    collaborator NIH

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

    collaborator NIH

  • Lysosomal Disease Network

    collaborator OTHER

  • University of Minnesota

    lead OTHER

Principal Investigators

  • Chester B. Whitley, M.D., Ph.D. · University of Minnesota

  • Ashley Schneider · University of Minnesota

  • Paul Harmatz, M.D. · Oakland Children's Hospital

  • Michal Inbar-Feigenberg, M.D. · Hospital for Sick Children, Toronto, Ontario, CA

  • Heather Lau, M.D. · New York University

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-09-30
Primary Completion
2019-08-31
Completion
2019-08-31

Countries

  • United States
  • Canada

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01870375 on ClinicalTrials.gov