New drug DYNE-101 aims to ease muscle symptoms in DM1
NCT ID NCT07486934
First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 3 times
Summary
This Phase 3 trial tests whether DYNE-101 can improve muscle function and daily life in 150 adults with myotonic dystrophy type 1 (DM1). Participants receive either the drug or a placebo by IV every few weeks for 48 weeks. The study measures how quickly people can stand from a chair, walk, and open their hands, along with overall health and safety.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
zeleciment basivarsen (DYNE-101)
What this could lead to
If successful, this could lead to a treatment that improves muscle function and daily living for people with myotonic dystrophy type 1.
What could go wrong
This is a Phase 3 trial, but it is still experimental. The drug may not work better than placebo, and there could be side effects from the intravenous infusions.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for DM1 are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit
RECRUITINGBerlin, State of Berlin, 13125, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Hospital Universitario Infanta Sofía
RECRUITINGSan Sebastián de los Reyes, Madrid, 28702, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
IU Health Neuroscience Center
RECRUITINGIndianapolis, Indiana, 46202, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus Innenstadt
RECRUITINGMunich, Bavaria, 80336, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
National Hospital Organization Osaka Toneyama Medical Center
RECRUITINGToyonaka-Shi, Osaka, 560-8552, Japan
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Pitié-Salpêtrière Hospital
RECRUITINGParis, 75013, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Rare Disease Research, LLC
RECRUITINGAtlanta, Georgia, 30329, United States
Contact Email: •••••@•••••
-
Rare Disease Research, LLC
RECRUITINGHillsborough, North Carolina, 27278, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077)
RECRUITINGCopenhagen, 2100, Denmark
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Roy Blunt NextGen Precision Health Institute
RECRUITINGColumbia, Missouri, 65211, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
The University of Texas Health Science Center at San Antonio
RECRUITINGSan Antonio, Texas, 78229, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
UZ Leuven
RECRUITINGLeuven, 3000, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery
RECRUITINGLondon, England, WC1N 3BG, United Kingdom
Contact Email: •••••@•••••
-
Yamaguchi University Hospital
RECRUITINGUbe-Shi, Yamaguchi, 755-8505, Japan
Contact Email: •••••@•••••