Actively Recruiting
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Led by Virginia Commonwealth University · Updated on 2025-06-06
700
Participants Needed
17
Research Sites
413 weeks
Total Duration
On this page
Sponsors
V
Virginia Commonwealth University
Lead Sponsor
U
University of Rochester
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
CONDITIONS
Official Title
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 to 70 (inclusive)
- Able to provide informed consent
- Clinical diagnosis of DM1 based on research criteria or positive genetic test
- Muscle biopsy sub-study participants must have at least moderate ankle muscle weakness (MRC score 64 4+)
You will not qualify if you...
- Symptomatic kidney or liver disease, uncontrolled diabetes or thyroid disorder, active cancer other than skin cancer
- Current alcohol or substance abuse
- Enrolled in another DM1 clinical trial or participated within 6 months
- Pregnant or planning pregnancy during the study
- Medical conditions that could affect study performance as judged by investigators
- For muscle biopsy sub-study: CTG repeat size less than 100 repeats without clear limb weakness and muscle wasting
- Use of anticoagulants like warfarin or direct oral anticoagulants
- Use of aspirin or NSAIDs not stopped 3 days before biopsy if possible
- Platelet count under 50,000 if known
- History of bleeding disorders
- Severe wasting of tibialis anterior muscle preventing biopsy
- Previous muscle biopsy of tibialis anterior muscle
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 17 locations
1
University of California, San Diego
La Jolla, California, United States, 92703
Actively Recruiting
2
University of California, Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
3
University of Colorado - Denver
Denver, Colorado, United States, 80204
Actively Recruiting
4
University of Florida
Gainesville, Florida, United States, 32611
Actively Recruiting
5
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
6
Kansas University Medical Center
Kansas City, Kansas, United States, 66160
Actively Recruiting
7
University of Rochester
Rochester, New York, United States, 14642
Actively Recruiting
8
Ohio State University
Columbus, Ohio, United States, 43210
Actively Recruiting
9
Houston Methodist Neurological Institute
Houston, Texas, United States, 77030
Actively Recruiting
10
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Actively Recruiting
11
Université de Sherbrooke
Québec, Canada
Active, Not Recruiting
12
Friedrich Baur Institute, Ludwig-Maximilians-Universität München
München, Germany
Actively Recruiting
13
Centro Clinico NeMO
Milan, Italy
Completed
14
Radboud University Medical Center
Nijmegen, Netherlands
Actively Recruiting
15
University of Auckland
Auckland, New Zealand
Actively Recruiting
16
St. George's, University of London
London, United Kingdom
Actively Recruiting
17
University College London
London, United Kingdom
Actively Recruiting
Research Team
J
Jennifer Raymond
CONTACT
R
Ruby Langeslay
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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