Clinical trialNCT03981575RecruitingObservational

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

SponsorVirginia Commonwealth UniversityEnrollment700DM1Myotonic Dystrophy 1

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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

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Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Charles Thornton, MD
University of Rochester
Principal Investigator
clinicaltrials.gov ↗
Nicholas Johnson, MD
Virginia Commonwealth University
Principal Investigator
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

17 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Canada

Université de Sherbrooke
Québec, Canada
Active, not recruiting

Germany

Friedrich Baur Institute, Ludwig-Maximilians-Universität München
München, Germany
Benedikt Schoser, FEAN
Recruiting

Italy

Centro Clinico Nemo
Milan, Italy
Completed

Netherlands

Radboud University Medical Center
Nijmegen, Netherlands
Baziel van Engelen, MD
Recruiting

New Zealand

University of Auckland
Auckland, New Zealand
Richard Roxburgh
Recruiting

United Kingdom

St. George'S, University of London
London, United Kingdom
Emma Matthews
Recruiting
University College London
London, United Kingdom
Chris Turner
Recruiting

United States

University of California, San Diego
La Jolla, California, United States
Chamindra Laverty, MD
Recruiting
University of California, Los Angeles
Los Angeles, California, United States
Perry Shieh, MD, PhD
Recruiting
University of Colorado - Denver
Denver, Colorado, United States
Matthew Wicklund, MD
Recruiting
University of Florida
Gainesville, Florida, United States
Sankarsubramoney Subramony, MD
Recruiting
University of Iowa
Iowa City, Iowa, United States
Andrea Swenson, MD
Recruiting
Kansas University Medical Center
Kansas City, Kansas, United States
Jeffrey Statland, MD
Recruiting
University of Rochester
Rochester, New York, United States
Johanna Hamel, MD
Recruiting
Ohio State University
Columbus, Ohio, United States
Bakri Elsheikh, MD
Recruiting
Houston Methodist Neurological Institute
Houston, Texas, United States
Erika P. Greene
Recruiting
Virginia Commonwealth University
Richmond, Virginia, United States
Nicholas Johnson, MD
Recruiting

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study. It may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 1 Jan 2019
Primary completion: 1 Oct 2026
Completion: 1 Dec 2026
Last update posted: 10 Jun 2026
Registered enrollment: 700
Registry id: NCT03981575

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.