Evaluating the safety and tolerability of PGN-EDODM1 in patients with Myotonic Dystrophy Type 1
A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1 (FREEDOM-DM1)
This study is testing if a new treatment called PGN-EDODM1 is safe and well-tolerated for people with Myotonic Dystrophy Type 1 compared to a placebo.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 32 (estimated) |
| Ages | 18 Years to 50 Years |
| Sex | All |
| Sponsor | PepGen Inc Industry-sponsored |
| Locations | 12 sites (Irvine, California and 11 other locations) |
| Trial ID | NCT06204809 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the safety and tolerability of a single intravenous dose of PGN-EDODM1 in individuals diagnosed with Myotonic Dystrophy Type 1. Participants will undergo a screening period of up to 30 days followed by a 16-week treatment and observation period. The study will compare the effects of PGN-EDODM1 against a placebo to determine its safety profile and pharmacokinetics. The primary focus is on patients with a confirmed genetic diagnosis and specific muscle function criteria.
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 capabilities.
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 treatment could provide a new therapeutic option for patients suffering from Myotonic Dystrophy Type 1.
How similar studies have performed: While there have been studies on treatments for Myotonic Dystrophy, this specific approach with PGN-EDODM1 is novel and has not been extensively tested.
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 * Medical Research Council (MRC) score of ≥ Grade 4 in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner) * Presence of myotonia 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 * Medications specific for the treatment of myotonia within 2 weeks prior to screening * Percent predicted forced vital capacity (FVC) \<40% Note: Other inclusion and exclusion criteria may apply.
Where this trial is running
Irvine, California and 11 other locations
- UCI Center for Clinical Research — Irvine, California, United States (Recruiting)
- Stanford University — Palo Alto, California, United States (Recruiting)
- Rare Disease Research — Atlanta, Georgia, United States (Recruiting)
- University of Kansas Medical Center — Fairway, Kansas, United States (Recruiting)
- Massachusetts General Hospital — Boston, Massachusetts, United States (Recruiting)
- University of Rochester Medical Center — Rochester, New York, United States (Recruiting)
- Virginia Commonwealth University — Richmond, Virginia, United States (Recruiting)
- 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)
- University College London Hospital — London, UK, United Kingdom (Recruiting)
- Salford Royal Hospital — Salford, 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.