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

An Open-Label Extension Study Evaluating Safety and Pharmacokinetics in Participants With Myotonic Dystrophy Type 1 (FREEDOM-OLE)

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07220603

Enrollment

Registered

2025-10-24

Start date

2025-12-23

Completion date

2029-01-01

Last updated

2026-03-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Brief 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.

Interventions

DRUGPGN-EDODM1

Administered by intravenous (IV) infusion

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Healthy volunteers

Inclusion criteria

* Participant has completed a prior study with PGN-EDODM1

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

Design outcomes

Primary

Measure	Time frame
Safety and tolerability as assessed by number of participants with Adverse Events (AEs)	Baseline through Week 108

Countries

Canada

Contacts

CONTACTPepGen Patient Advocacy

clinicaltrials@pepgen.com781-797-0979

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 31, 2026