Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Recruiting in Palo Alto (17 mi)
+21 other locations
Overseen ByNicholas Johnson, MD
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Academic
Recruiting
Sponsor: Virginia Commonwealth University
No Placebo Group
Trial Summary
What is the purpose of this trial?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
Eligibility Criteria
Inclusion Criteria
Age 18 to 70 (inclusive)
Competent to provide informed consent
Clinical diagnosis of DM1 based on research criteria1 or positive genetic test
+1 more
Participant Groups
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
University of RochesterRochester, NY
Houston Methodist Neurological InstituteHouston, TX
Virginia Commonwealth UniversityRichmond, VA
University of California, Los AngelesLos Angeles, CA
More Trial Locations
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Who Is Running the Clinical Trial?
Virginia Commonwealth UniversityLead Sponsor
University College London HospitalsCollaborator
Institut de Myologie, FranceCollaborator
Ludwig-Maximilians - University of MunichCollaborator
Fondazione Serena Onlus - Centro Clinico NeMO MilanoCollaborator
University of RochesterCollaborator
The Methodist Hospital Research InstituteCollaborator
University of KansasCollaborator
Stanford UniversityCollaborator
Ohio State UniversityCollaborator