Active Studies

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

·         Study Status: Active, closed to enrollment

·         Patient Population: DM1

·         Participation Duration: 24 months

·         Active Sites:

o   Ludwig Maximilian University of Munich

o   NEuroMuscular Omnicentre

o   Radboud University

o   Stanford University

o   Virginia Commonwealth University

o   University College London

o   The University of Auckland

o   University of California, Los Angeles

o   University of Colorado

o   University of Florida Health

o   University of Iowa

o   University of Kansas Medical Center

o   University of Rochester Medical Center

o   St. George’s, University of London

o   Université de Sherbrooke

o   University of California, San Diego

o   Ohio State University

o   Houston Methodist Neurological Institute

o   Aomori National Hospital

o   University of Texas, San Antonio

o   University of Pennsylvania

·         Study Summary:

This is a non-interventional study observing the natural progression of DM1 in adult patients over the course of 24 months. The study’s primary goal is to identify standardized clinical outcome assessments, in the form of functional measures, to hasten therapeutic development.

Assessing Pediatric Endpoints in DM1 (ASPIRE-DM1)

·         Study Status: Open to enrollment

·         Patient Population:

o   CDM

·         Participant Duration: 18 months

·         Active Sites:

o   Virginia Commonwealth University

o   NEuroMuscular Omnicentre

o   University of California, Los Angeles

o   University of Kansas Medical Center

o   University of Rochester Medical Center

·         Study Summary:

This is a non-interventional study observing the natural progression of CDM in patients over the course of 18 months. The study’s primary goal is to evaluate appropriate measures of physical function, cognitive function, and quality of life in children with CDM .

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension (TREAT-EXT)

  • Study Status: Open to enrollment

  • Patient Population: CDM, ChDM

  • Participant Duration: 24 months

  • Active Sites:

    • Virginia Commonwealth University

  • Upcoming Sties:

    • NEuroMuscular Omnicentre Milano

    • University of Arkansas for Medical Sciences

    • University of California, San Diego

    • University of Kansas

    • University of Minnesota

    • University of Pennsylvania

    • University of Sao Paulo

  • Study Summary: This is a non-interventional study observing the natural progression of children with CDM and ChDM over the course of 24 months. The study’s primary goal is to evaluate appropriate clinical outcome assessments for older children with pediatric onset DM.