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

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.

Recruiting

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Key information

Conditions

Myotonic Dystrophy 1 Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonic Disorders Myotonic Dystrophy Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of Calgary, Calgary, Alberta, Canada

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

PGN-EDODM1

Drug

Administered by intravenous (IV) infusion

Primary outcomes

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

Time frame: Baseline through Week 108

Study contacts

Contact information is provided by the study sponsor or research team.

PepGen Patient Advocacy

CONTACT

[email protected]

781-797-0979

Sponsors and collaborators

Lead sponsor

PepGen Inc

Industry

Registry information

Official study title

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

Important dates

Study start: 2025
Primary completion: 2029
Study completion: 2029
First posted: Oct 24, 2025
Registry last updated: Mar 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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Completed