Biomarkers for Myotonic Dystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to better understand Myotonic Dystrophy Type 1 (DM1), a condition that causes muscle weakness and stiffness. Researchers seek reliable signs (biomarkers) and meaningful outcomes (endpoints) to track the disease. By examining patient differences and refining methods, they hope to improve how DM1 is studied and managed. Individuals diagnosed with DM1 who experience noticeable muscle issues may be suitable for this study. As an unphased trial, this study offers participants the opportunity to contribute to foundational research that could lead to better management and treatment of DM1.
Do I need to stop my current medications for the trial?
The trial does not specify if you need to stop taking your current medications, but if you are participating in the muscle biopsy sub-study, you should stop taking aspirin or non-steroidal anti-inflammatory agents 3 days before the procedure, if possible.
Why are researchers excited about this trial?
Researchers are excited about this trial because it aims to establish biomarkers and clinical endpoints for Myotonic Dystrophy Type 1 (DM1). Unlike current treatments that primarily focus on managing symptoms, this approach seeks to identify specific biological markers and measurable outcomes. This could lead to a better understanding of the disease progression and more precise ways to evaluate new treatments in the future. By identifying these key indicators, researchers hope to develop more targeted and effective therapies for DM1, ultimately improving patient care and outcomes.
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