New hope for DM1: Long-Term safety trial of PGN-EDODM1 now recruiting
NCT ID NCT07220603
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study is testing the long-term safety of an experimental drug called PGN-EDODM1 in 48 adults with myotonic dystrophy type 1 (DM1) who have already taken the drug in a previous study. Participants will receive the drug by IV infusion and be monitored for side effects. The goal is to see if the drug remains safe and tolerable over a longer period.
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 (given by IV infusion)
What this could lead to
If successful, this study could support the continued development of PGN-EDODM1 as a treatment to control myotonic dystrophy type 1 symptoms.
What could go wrong
This is an early-phase, open-label extension study with no placebo group, so results are preliminary. The drug may still fail in larger trials or cause unexpected side effects.
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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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CIUSSS du Saguenay-Lac-Saint-Jean
RECRUITINGSaguenay, Canada
Contact Email: •••••@•••••
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Ottawa Hospital Research Institute
RECRUITINGOttawa, Ontario, Canada
Contact Email: •••••@•••••
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University of Calgary
RECRUITINGCalgary, Alberta, T2N 4Z6, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••