New hope for muscle stiffness: experimental drug PGN-EDODM1 enters Mid-Stage trial
NCT ID NCT06667453
First seen Jun 24, 2026 · Last updated Jul 30, 2026 · Updated 3 times
Summary
This study tests an investigational drug called PGN-EDODM1 in 24 adults with myotonic dystrophy type 1, a condition causing muscle stiffness and weakness. Participants receive multiple doses of the drug or a placebo by IV to check safety and how the body processes it. The goal is to see if the drug is safe and tolerable.
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 successful, this could point toward a treatment that controls symptoms of myotonic dystrophy type 1, like muscle stiffness and weakness.
- What could go wrong
- This is an early Phase 2 trial with only 24 participants, so results may not apply to everyone. The drug is still experimental and may not work or could have side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for MYOTONIC DYSTROPHY 1 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
Email: •••••@•••••
Locations
-
CIUSSS du Saguenay-Lac-Saint-Jean
RECRUITINGChicoutimi, Quebec, Canada
-
Cardiff and Vale Hospital
RECRUITINGCardiff, United Kingdom
Contact Email: •••••@•••••
-
Kyungpook National University Chilgok Hospital
RECRUITINGDaegu, South Korea
-
Montreal Neurological Institute
RECRUITINGMontreal, Quebec, H3A 2B4, Canada
Contact Email: •••••@•••••
-
Newcastle Upon Tyne Hospitals
RECRUITINGNewcastle upon Tyne, NE7 7DN, United Kingdom
Contact Email: •••••@•••••
-
Ottawa Hospital Research Institute (OHRI)
RECRUITINGOttawa, Ontario, Canada
-
Pacific Clinical Research Network Auckland
RECRUITINGTakapuna, Auckland, 0622, New Zealand
-
Pusan National University Yangsan Hospital
RECRUITINGPusan, Gyeongsangnam-do, South Korea
Contact Email: •••••@•••••
-
Salford Royal Hospital
RECRUITINGSalford, England, United Kingdom
Contact Email: •••••@•••••
-
Seoul National University Hospital
RECRUITINGSeoul, South Korea
-
University College London Hospitals NHS Foundation Trust
RECRUITINGLondon, UK, United Kingdom
Contact Email: •••••@•••••
-
University of Calgary
RECRUITINGCalgary, Alberta, T2N 4Z6, Canada
Contact Email: •••••@•••••
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can watching the course of myotonic dystrophy unlock better care?
- Scientists hunt for biomarkers to unlock DM1 treatments
- 700-Patient study seeks key clues to muscle disease
- Brain scans reveal diabetes link to cognitive decline in rare disease
- New drug AOC 1001 tested for rare muscle disease
- Spanish researchers launch massive DM1 registry to unlock disease secrets