Summary

Eligibility
for people ages 18-70 (full criteria)
Location
at UCLA UCSD
Dates
study started
completion around
Principal Investigator
by Chamindra Laverty, MD (ucsd)Perry Shieh, MD, PhD (ucla)

Description

Summary

Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogeneity going forward.

Funding Source- FDA OOPD

Official Title

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Details

Keywords

Myotonic Dystrophy 1, DM1, Myotonic Dystrophy, END DM-1, Muscular Dystophy, DMCRN, Study Visits

Eligibility

Locations

  • University of California, San Diego accepting new patients
    La Jolla California 92703 United States
  • University of California, Los Angeles accepting new patients
    Los Angeles California 90095 United States
  • Stanford University in progress, not accepting new patients
    Stanford California 94305 United States

Lead Scientists at University of California Health

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Virginia Commonwealth University
ID
NCT03981575
Study Type
Observational
Participants
Expecting 700 study participants
Last Updated