Trials / Completed
CompletedNCT06204809
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants 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)
- Status
- Completed
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 24 (actual)
- Sponsor
- PepGen Inc · Industry
- Sex
- All
- Age
- 18 Years – 60 Years
- Healthy volunteers
- Not accepted
Summary
The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | PGN-EDODM1 for infusion | Single dose of PGN-EDODM1 by intravenous (IV) infusion |
| OTHER | Placebo | Administered by IV infusion |
Timeline
- Start date
- 2023-12-12
- Primary completion
- 2025-10-28
- Completion
- 2025-10-28
- First posted
- 2024-01-12
- Last updated
- 2026-02-13
Locations
12 sites across 3 countries: United States, Canada, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06204809. Inclusion in this directory is not an endorsement.