A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (NCT06667453)
A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
๐ฉบ Plain-English Summary (8th-Grade Level)
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.
Who can join: Review the eligibility criteria below.
Location: University of Calgary โ Calgary, Alberta
Age range: 16 Years to 65 Years
๐งช Interventions in This Study
๐ "Do I Qualify?" โ 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria โ
๐ All Participating Trial Locations (12 Sites)
- University of Calgary โ Calgary, Alberta
- Ottawa Hospital Research Institute (OHRI) โ Ottawa, Ontario
- CIUSSS du Saguenay-Lac-Saint-Jean โ Chicoutimi, Quebec
- Montreal Neurological Institute โ Montreal, Quebec
- Pacific Clinical Research Network Auckland โ Takapuna, Auckland
- Pusan National University Yangsan Hospital โ Pusan, Gyeongsangnam-do
- Kyungpook National University Chilgok Hospital โ Daegu,
- Seoul National University Hospital โ Seoul,
- Salford Royal Hospital โ Salford, England
- University College London Hospitals NHS Foundation Trust โ London, UK
- Cardiff and Vale Hospital โ Cardiff,
- Newcastle Upon Tyne Hospitals โ Newcastle upon Tyne,
๐ Contact the Study Coordinator
Phone: (781) 797-0979
Email: clinicaltrials@pepgen.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
๐ฐ Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Myotonic Dystrophy 1
- Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy โ Liverpool, New South Wales
- Study of ATX-01 in Participants With DM1 โ Los Angeles, California
- Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) โ La Jolla, California
- An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE) โ Calgary, Alberta
Explore by condition: All Myotonic Dystrophy 1 clinical trials
๐ก How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.