Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
What You Need to Know Before You Apply
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
Who Is on the Research Team?
Nicholas Johnson, MD
Principal Investigator
Virginia Commonwealth University
Charles Thornton, MD
Principal Investigator
University of Rochester
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Baseline Assessment
Initial assessment and data collection at the start of the study
Longitudinal Study
Participants undergo regular assessments to track disease progression and collect data over time
Muscle Biopsy Sub-study
A subset of participants undergo muscle biopsy to study splicing biomarkers
Follow-up
Participants are monitored for safety and effectiveness after the main study period
What Are the Treatments Tested in This Trial?
Interventions
- Establishing Biomarkers and Clinical Endpoints
Find a Clinic Near You
Who Is Running the Clinical Trial?
Virginia Commonwealth University
Lead Sponsor
University College London Hospitals
Collaborator
Institut de Myologie, France
Collaborator
Ludwig-Maximilians - University of Munich
Collaborator
Fondazione Serena Onlus - Centro Clinico NeMO Milano
Collaborator
University of Rochester
Collaborator
The Methodist Hospital Research Institute
Collaborator
University of Kansas
Collaborator
Stanford University
Collaborator
Ohio State University
Collaborator