700 Participants Needed

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

Recruiting at 21 trial locations
JS
NJ
JR
RL
JR
Overseen ByJennifer Raymond
Age: 18+
Sex: Any
Trial Phase: Academic
Sponsor: Virginia Commonwealth University
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

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?

NJ

Nicholas Johnson, MD

Principal Investigator

Virginia Commonwealth University

CT

Charles Thornton, MD

Principal Investigator

University of Rochester

Are You a Good Fit for This Trial?

Inclusion Criteria

Age 18 to 70 (inclusive)
Competent to provide informed consent
Clinical diagnosis of DM1 based on research criteria1 or positive genetic test
See 1 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Baseline Assessment

Initial assessment and data collection at the start of the study

0 months
1 visit (in-person)

Longitudinal Study

Participants undergo regular assessments to track disease progression and collect data over time

24 months
3 visits (in-person) at 0, 12, and 24 months

Muscle Biopsy Sub-study

A subset of participants undergo muscle biopsy to study splicing biomarkers

3 months
1 additional visit (in-person) for biopsy

Follow-up

Participants are monitored for safety and effectiveness after the main study period

4 weeks

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

Trials
732
Recruited
22,900,000+

University College London Hospitals

Collaborator

Trials
204
Recruited
1,221,000+

Institut de Myologie, France

Collaborator

Trials
31
Recruited
6,600+

Ludwig-Maximilians - University of Munich

Collaborator

Trials
365
Recruited
210,000+

Fondazione Serena Onlus - Centro Clinico NeMO Milano

Collaborator

Trials
8
Recruited
6,300+

University of Rochester

Collaborator

Trials
883
Recruited
555,000+

The Methodist Hospital Research Institute

Collaborator

Trials
299
Recruited
82,500+

University of Kansas

Collaborator

Trials
157
Recruited
332,000+

Stanford University

Collaborator

Trials
2,527
Recruited
17,430,000+

Ohio State University

Collaborator

Trials
891
Recruited
2,659,000+
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