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

Summary

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

• Myotonic Dystrophy 1
• DM1

Sponsors & Collaborators

• University of Rochester

  collaborator OTHER

• Stanford University

  collaborator OTHER

• Ohio State University

  collaborator OTHER

• University of Florida

  collaborator OTHER

• University of Iowa

  collaborator OTHER

• Ludwig-Maximilians - University of Munich

  collaborator OTHER

• Fondazione Serena Onlus - Centro Clinico NeMO Milano

  collaborator OTHER

• The Methodist Hospital Research Institute

  collaborator OTHER

• Radboud University Medical Center

  collaborator OTHER

• University College London Hospitals

  collaborator OTHER

• University of California, Los Angeles

  collaborator OTHER

• Virginia Commonwealth University

  lead OTHER

Principal Investigators

• Nicholas Johnson, MD · Virginia Commonwealth University

• Charles Thornton, MD · University of Rochester

Eligibility

Min Age

18 Years

Max Age

70 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2019-01-01

Primary Completion

2026-10-01

Completion

2026-12-01

Countries

• United States
• Canada
• Germany
• Italy
• Netherlands
• New Zealand
• United Kingdom

Study Locations