NCT06667453 A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
| NCT ID | NCT06667453 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | PepGen Inc |
| Condition | Myotonic Dystrophy 1 |
| Study Type | INTERVENTIONAL |
| Enrollment | 24 participants |
| Start Date | 2024-12-10 |
| Primary Completion | 2027-03 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 24 participants in total. It began in 2024-12-10 with a primary completion date of 2027-03.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: * Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats * Presence of myotonia * Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed * Body Mass Index (BMI) of \< 35.0 kg/m\^2 Exclusion Criteria: * Congenital DM1 * Known history or presence of any clinically significant conditions that may interfere with study safety assessments * Abnormal laboratory tests at screening considered clinically significant by the Investigator * Medications specific for the treatment of myotonia within 2 weeks prior to screening * Percent predicted forced vital capacity (FVC) \<40% * Use of an investigational drug, device, or product within 30 days of 5 half-lives of the study drug (whichever is longer) prior to Screening Note: Other inclusion and exclusion criteria may apply.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06667453 clinical trial?
This trial is open to participants of all sexes, aged 16 Years or older, up to 65 Years, studying Myotonic Dystrophy 1. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06667453 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT06667453 currently recruiting?
Yes, NCT06667453 is actively recruiting participants. Contact the research team at clinicaltrials@pepgen.com for enrollment information.
Where is the NCT06667453 trial being conducted?
This trial is being conducted at Calgary, Canada, Ottawa, Canada, Chicoutimi, Canada, Montreal, Canada and 8 additional locations.
Who is sponsoring the NCT06667453 clinical trial?
NCT06667453 is sponsored by PepGen Inc. The trial plans to enroll 24 participants.