New drug PGN-EDODM1 tested for muscle disease
NCT ID NCT06204809
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tested a single dose of the drug PGN-EDODM1 in 24 adults with myotonic dystrophy type 1, a genetic muscle disorder. The main goal was to check safety and how the body handles the drug. The study is complete, but results are not yet available.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
PGN-EDODM1
What this could lead to
If safe, this could pave the way for a treatment that targets the root cause of myotonic dystrophy type 1.
What could go wrong
This is an early Phase 1 safety trial with only 24 people. It does not yet test if the drug works, and side effects are unknown.
Disclaimer
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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.
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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
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Locations
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CIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi, Quebec, Canada
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Ottawa Hospital Research Institute (OHRI)
Ottawa, Ontario, Canada
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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Salford Royal Hospital
Salford, United Kingdom
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Stanford University
Palo Alto, California, 94304, United States
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UCI Center for Clinical Research
Irvine, California, 92697, United States
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University College London Hospital
London, UK, NW1 2PG, United Kingdom
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University of Calgary
Calgary, Alberta, T3M 1M4, Canada
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University of Kansas Medical Center
Fairway, Kansas, 66205, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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Virginia Commonwealth University
Richmond, Virginia, 23298, United States