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CompletedNCT06204809FREEDOM-DM1Updated Feb 13, 2026

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

A Phase 1 interventional study of PGN-EDODM1 for infusion and Placebo in Myotonic Dystrophy 1, sponsored by PepGen Inc. Completed at 12 sites in 3 countries. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2026-02-13.

Sponsored by PepGen Inc · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
24
Allocation
Randomized
Ages
18 Years to 60 Years
Sex
All
01

Study 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).

02

Conditions studied

  • Myotonic Dystrophy 1

Keywords

  • Myotonic Dystrophy
  • Muscular Dystrophies
  • Genetic Diseases, Inborn
  • Neuromuscular Diseases
  • Nervous System Diseases
  • Musculoskeletal Diseases
  • Myotonic Disorders
  • Muscular Disorders, Atrophic
  • Heredodegenerative Disorders, Nervous System
  • Neurodegenerative Diseases
  • Muscular Diseases
  • Steinert Disease
03

Who can participate

Ages eligible
18 Years to 60 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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%

Note: Other inclusion and exclusion criteria may apply.

04

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
24 participants (actual)

Study arms

  • Experimental
    PGN-EDODM1

    PGN-EDODM1 for infusion

    Drug: PGN-EDODM1 for infusion

  • Placebo comparator
    Placebo

    0.9% NaCl

    Other: Placebo

Interventions

  • DrugPGN-EDODM1 for infusion

    Single dose of PGN-EDODM1 by intravenous (IV) infusion

  • OtherPlacebo

    Administered by IV infusion

05

What researchers measure

Primary outcomes

  1. Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs

    Time frame: Baseline to Week 16

Secondary outcomes

  1. Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1

    Time frame: Baseline up to Day 3

  2. Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1

    Time frame: Baseline up to Day 3

  3. Apparent Terminal Half-Life (t½) of PGN-EDODM1

    Time frame: Baseline up to Day 3

  4. Area Under the Concentration-time Curve of PGN-EDODM1

    Time frame: Baseline up to Day 3

06

Study locations

12 sites
  • UCI Center for Clinical Research
    Irvine, California 92697, United States
  • Stanford University
    Palo Alto, California 94304, United States
  • Rare Disease Research
    Atlanta, Georgia 30329, United States
  • University of Kansas Medical Center
    Fairway, Kansas 66205, United States
  • Massachusetts General Hospital
    Boston, Massachusetts 02114, United States
  • University of Rochester Medical Center
    Rochester, New York 14642, United States
  • Virginia Commonwealth University
    Richmond, Virginia 23298, United States
  • University of Calgary
    Calgary, Alberta T3M 1M4, Canada
  • Ottawa Hospital Research Institute (OHRI)
    Ottawa, Ontario, Canada
  • CIUSSS du Saguenay-Lac-Saint-Jean
    Chicoutimi, Quebec, Canada
  • University College London Hospital
    London, UK NW1 2PG, United Kingdom
  • Salford Royal Hospital
    Salford, United Kingdom
07

Registry details

Key details

Study ID
NCT06204809
Lead sponsor
PepGen Inc
Responsible party
Sponsor
First posted
Jan 12, 2024
Start date
Dec 12, 2023
Primary completion
Oct 28, 2025
Completion
Oct 28, 2025
Last update
Feb 13, 2026

Oversight

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

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