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RecruitingNCT07220603Updated Mar 30, 2026

An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)

A Phase 2 interventional study of PGN-EDODM1 in Myotonic Dystrophy 1, sponsored by PepGen Inc. Recruiting at 3 sites in Canada. Per ClinicalTrials.gov, last updated 2026-03-30.

Sponsored by PepGen Inc · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
48
Allocation
Not applicable
Sex
All
01

Study summary

The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1.

02

Conditions studied

  • Myotonic Dystrophy 1

Keywords

  • DM1
  • Myotonic Dystrophy 1
  • Myotonic Dystrophy
  • PepGen
  • PGN-EDODM1
  • Myotonic Muscular Dystrophy
  • Steinhert's Disease
  • Myotonic Dystrophies
  • Genetic Diseases, Inborn
  • Neuromuscular Diseases
  • Nervous System Diseases
  • Musculoskeletal Diseases
  • Myotonic Disorders
  • Muscular Disorders, Atrophic
  • Heredodegenerative Disorders, Nervous System
  • Muscular Diseases
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Participant has completed a prior study with PGN-EDODM1

Exclusion criteria

Exclusion Criteria:

  • Abnormal laboratory tests at screening considered clinically significant by the Investigator
  • Use of an investigational drug (other than PGN-EDODM1), device, or product, within 30 days or 5 half-lives of the study drug (whichever is longer) prior to study entry
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
48 participants (estimated)

Study arms

  • Experimental
    PGN-EDODM1

    Participants will receive doses of PGN-EDODM1 once every 4 weeks (Q4W)

    Drug: PGN-EDODM1

Interventions

  • DrugPGN-EDODM1

    Administered by intravenous (IV) infusion

05

What researchers measure

Primary outcomes

  1. Safety and tolerability as assessed by number of participants with Adverse Events (AEs)

    Time frame: Baseline through Week 108

06

Study locations

3 of 3 sites recruiting
07

Registry details

Key details

Study ID
NCT07220603
Lead sponsor
PepGen Inc
Responsible party
Sponsor
First posted
Oct 24, 2025
Start date
Dec 23, 2025
Primary completion
Jan 2029 (estimated)
Completion
Jan 2029 (estimated)
Last update
Mar 30, 2026

Study contacts

PepGen Patient Advocacy
Contact
clinicaltrials@pepgen.com
781-797-0979

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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