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Tracking Neurodegeneration in Early Wolfram Syndrome

NCT02455414 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 101

Last updated 2018-07-11

No results posted yet for this study

Summary

The goal of this study is to determine the pattern of early neurodegenerative changes in WFS (Wolfram Syndrome). The investigator will perform cross-sectional and longitudinal assessments of youth with WFS, targeting sensitive neural systems with quantified neuroimaging and behavioral measures. In addition, the investigator will establish the utility of a WFS severity rating scale (WFS Unified Rating Scale or WURS). Preliminary data support the feasibility of this approach and its potential to generate important new information about neurodevelopmental and neurodegenerative patterns in WFS. This work is necessary to position the field for future clinical trials to test interventions for WFS neurodegeneration. Ultimately, a better understanding of the trajectory of neurodegeneration in WFS and the development of effective interventions may be relevant to other more common neurodegenerative and endocrine (Type 1 and Type 2 diabetes) diseases in which ER stress has been implicated.

Conditions

Sponsors & Collaborators

  • National Institutes of Health (NIH)

    collaborator NIH

  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

    collaborator NIH

  • Washington University School of Medicine

    lead OTHER

Principal Investigators

  • Tamara Hershey, PhD · Washington University Medical School

Eligibility

Min Age
1 Day
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2012-04-30
Primary Completion
2017-07-12
Completion
2017-07-12

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

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 NCT02455414 on ClinicalTrials.gov