NCT06204809: FREEDOM-DM1

Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

Completed Phase 1 Last updated 13 February 2026

What this trial tests

Phase 1 trial testing PGN-EDODM1 for infusion in Myotonic Dystrophy 1 in 24 participants. Completed in 28 October 2025.

Timeline

12 December 2023

Primary endpoint
28 October 2025

28 October 2025

Quick facts

Lead sponsor	PepGen Inc
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	24
Start date	12 December 2023
Primary completion	28 October 2025
Estimated completion	28 October 2025
Sites	12 locations across United Kingdom, Canada, United States

Drugs / interventions tested

• PGN-EDODM1 for infusion — full drug profile →
• Placebo

Conditions studied

• Myotonic Dystrophy 1 — all drugs for Myotonic Dystrophy 1 →

Sponsor

PepGen Inc — full company profile →

Who can join

Adults 18 to 60, any sex, with Myotonic Dystrophy 1. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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).

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

1. Evolution of antisense oligonucleotides: navigating nucleic acid chemistry and delivery challenges.
   Ruchi R, Raman GM, Kumar V, Bahal R. · · 2025 · cited 9× · PMID 39653607 · DOI 10.1080/17460441.2024.2440095
2. Antisense RNA therapies for muscular dystrophies.
   Arechavala-Gomeza V, López-Martínez A, Aartsma-Rus A. · · 2026 · cited 7× · PMID 40150900 · DOI 10.1177/22143602251324858
3. Multisystem Symptoms in Myotonic Dystrophy Type 1: A Management and Therapeutic Perspective.
   Kuntawala DH, Vitorino R, Cruz AC, Martins F, et al · · 2025 · cited 4× · PMID 40508159 · DOI 10.3390/ijms26115350
4. Inhibition of SARS-CoV-2 growth in the lungs of mice by a peptide-conjugated morpholino oligomer targeting viral RNA.
   Sakai A, Singh G, Khoshbakht M, Bittner S, et al · · 2024 · cited 4× · PMID 39376996 · DOI 10.1016/j.omtn.2024.102331
5. Analysis of human urinary extracellular vesicles reveals disordered renal metabolism in myotonic dystrophy type 1.
   Kumari P, Sullivan LM, Li Z, Parker Conquest E, et al · · 2025 · cited 1× · PMID 40044661 · DOI 10.1038/s41467-025-56479-5