Investigating PGN-EDODM1 for Myotonic Dystrophy Type 1
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)
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.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 24 (estimated) |
| Ages | 16 Years to 65 Years |
| Sex | All |
| Sponsor | PepGen Inc Industry-sponsored |
| Locations | 8 sites (Calgary, Alberta and 7 other locations) |
| Trial ID | NCT06667453 on ClinicalTrials.gov |
What this trial studies
This clinical study evaluates the safety and tolerability of an investigational medication, PGN-EDODM1, in individuals diagnosed with myotonic dystrophy type 1 (DM1). Participants will receive either PGN-EDODM1 or a placebo in multiple administrations to assess the drug's effects. The study aims to determine how well the medication is tolerated compared to a control group, focusing on individuals with specific muscle function criteria and genetic markers associated with DM1.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals with a confirmed diagnosis of myotonic dystrophy type 1 and specific muscle function criteria.
Not a fit: Patients with congenital myotonic dystrophy or those with significant comorbid conditions may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to a new treatment option for patients with myotonic dystrophy type 1, potentially improving their quality of life.
How similar studies have performed: While this approach is novel, similar studies targeting genetic conditions have shown promise in other contexts.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Calgary, Alberta and 7 other locations
- University of Calgary — Calgary, Alberta, Canada (Recruiting)
- Ottawa Hospital Research Institute (OHRI) — Ottawa, Ontario, Canada (Recruiting)
- CIUSSS du Saguenay-Lac-Saint-Jean — Chicoutimi, Quebec, Canada (Recruiting)
- Montreal Neurological Institute — Montreal, Quebec, Canada (Recruiting)
- Pacific Clinical Research Network Auckland — Takapuna, Auckland, New Zealand (Active_not_recruiting)
- Salford Royal Hospital — Salford, England, United Kingdom (Recruiting)
- University College London Hospitals NHS Foundation Trust — London, UK, United Kingdom (Recruiting)
- Newcastle Upon Tyne Hospitals — Newcastle upon Tyne, United Kingdom (Recruiting)
Study contacts
- Study coordinator: PepGen
- Email: clinicaltrials@pepgen.com
- Phone: 781-797-0979
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.