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Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 | Clinical Trials | Clareo Health

COMPLETEDPHASE1

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)

NCT06204809•PepGen Inc

View on ClinicalTrials.gov

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).

Eligibility

Age

18 - 60 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
•Medical Research Council (MRC) score of ≥ Grade 4- in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
•Presence of myotonia

Exclusion Criteria

•Congenital DM1
•Known history or presence of any clinically significant conditions that may interfere with study safety assessments
•Abnormal laboratory tests at screening
•Medications specific for the treatment of myotonia within 2 weeks prior to screening
•Percent predicted forced vital capacity (FVC) \<40%

Locations (12)

UCI Center for Clinical Research

Irvine, California, United States

Stanford University

Palo Alto, California, United States

Rare Disease Research

Atlanta, Georgia, United States

University of Kansas Medical Center

Fairway, Kansas, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

University of Rochester Medical Center

Rochester, New York, United States

Virginia Commonwealth University

Richmond, Virginia, United States

University of Calgary

Calgary, Alberta, Canada

Ottawa Hospital Research Institute (OHRI)

Ottawa, Ontario, Canada

CIUSSS du Saguenay-Lac-Saint-Jean

Chicoutimi, Quebec, Canada

Key Dates

Start Date

December 12, 2023

Primary Completion Date

October 28, 2025

Completion Date

October 28, 2025

Last Updated

February 13, 2026

Enrollment

ACTUAL participants

Conditions

Myotonic Dystrophy 1

Interventions

PGN-EDODM1 for infusion

DRUG

Placebo

OTHER

Study of ATX-01 in Participants With DM1

NCT06300307

Myotonic Dystrophy 1

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RECRUITING

Myotonic Dystrophy Family Registry

NCT02398786

Myotonic DystrophyCongenital Myotonic Dystrophy+14 more

View study

ENROLLING_BY_INVITATIONPHASE3

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 13, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

Study of ATX-01 in Participants With DM1

Myotonic Dystrophy Family Registry

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

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