Long-term PGN-EDODM1 extension for people with myotonic dystrophy type 1
Official title: An Open-Label Extension Study Evaluating Safety and Pharmacokinetics in Participants With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
Run by PepGen Inc · NCT07220603 · Data updated 2026-11-12
This open-label extension will try continued PGN-EDODM1 in people with myotonic dystrophy type 1 who completed a prior PGN-EDODM1 study to see how safe and tolerable it is over time.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Sex | All |
| Enrollment | 48 (estimated) |
| Sponsor | PepGen Inc Industry-sponsored |
| Locations | 3 sites (Calgary, Alberta and 2 other locations) |
| Status | Recruiting |
| Started | December 23, 2025 |
| Main results expected | January 2029 |
| Study ends | January 2029 |
| Registry record updated | March 30, 2026 |
| Trial ID | NCT07220603 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are people with myotonic dystrophy type 1 who completed a prior PGN-EDODM1 study and meet current screening labs and medication restrictions.
✕Probably not a fit
People who have not participated in a prior PGN-EDODM1 study, who have clinically significant abnormal screening labs, or who recently used other investigational products are unlikely to be eligible or benefit from this extension.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This open-label extension enrolls participants with myotonic dystrophy type 1 who previously completed a PGN-EDODM1 trial to collect longer-term safety and tolerability data. Participants will continue to receive PGN-EDODM1 and undergo regular clinical visits, laboratory testing, and adverse event monitoring. The protocol excludes people with clinically significant abnormal labs at screening or recent use of other investigational products. The study is conducted at multiple Canadian sites and focuses on safety outcomes rather than efficacy endpoints.
Why it matters
Potential benefit: If successful, the study could demonstrate that PGN-EDODM1 is safe to use long-term in people with DM1 and support continued development and access.
How similar studies have gone: Some related oligonucleotide and gene-targeting approaches in DM1 have shown early promise, but PGN-EDODM1 long-term data are limited and this extension is largely confirmatory for safety.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Participant has completed a prior study with PGN-EDODM1 Exclusion Criteria: * Abnormal laboratory tests at screening considered clinically significant by the Investigator * Use of an investigational drug (other than PGN-EDODM1), device, or product, within 30 days or 5 half-lives of the study drug (whichever is longer) prior to study entry
Where it's running
-
University of Calgary
Calgary, Alberta, Canada Recruiting -
Ottawa Hospital Research Institute
Ottawa, Ontario, Canada Recruiting -
CIUSSS du Saguenay-Lac-Saint-Jean
Saguenay, Canada Recruiting
Study contacts
- Study coordinator: PepGen Patient Advocacy
- Email: clinicaltrials@pepgen.com
- Phone: 781-797-0979
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
Understanding long-term progression in myotonic dystrophy type 1 (DM1)
This study will follow adults with DM1 for at least four years to see how walking, hand function, muscle strength, heart rhythms, and lung function change over time.
-
Spanish National Registry for Myotonic Dystrophy Type 1
This registry gathers health, clinical, and genetic information from people in Spain with DM1 to see how the condition changes over time and help match patients to future trials.
-
Wearable monitoring of walking and arm movement in adults with neuromuscular or metabolic movement disorders
This project uses a wrist/ankle wearable to continuously monitor walking and upper-limb movement in ambulant adults with neuromuscular diseases or obesity to see if digital measures like stride speed track real-life motor function.
-
Investigating PGN-EDODM1 for Myotonic Dystrophy Type 1
This study is testing a new medication called PGN-EDODM1 to see if it is safe and well-tolerated in people with myotonic dystrophy type 1.
-
Testing ATX-01 for myotonic dystrophy type 1
This study is testing a new drug called ATX-01 to see if it is safe and well-tolerated in people with myotonic dystrophy type 1.
-
Collecting data on muscle disorders using wearable devices
This study is testing a wearable device to track movement in people with muscle disorders like Duchenne Muscular Dystrophy and Myotonic Dystrophy to see how it can help improve their care.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.