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RecruitingNCT06667453FREEDOM2-DM1Updated Jul 29, 2026

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1

A Phase 2 interventional study of PGN-EDODM1 and Placebo in Myotonic Dystrophy 1, sponsored by PepGen Inc. Recruiting at 12 sites in 4 countries. Open to participants aged 16 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-07-29.

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

Phase
Phase 2
Study type
Interventional
Enrollment
24
Allocation
Randomized
Ages
16 Years to 65 Years
Sex
All
01

Study summary

The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.

02

Conditions studied

  • Myotonic Dystrophy 1

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Keywords

  • DM1
  • Myotonic Dystrophy 1
  • Myotonic Dystrophy
  • PepGen
  • PGN-EDODM1
  • Myotonic Muscular Dystrophy
  • Steinert's Disease
03

Who can participate

Ages eligible
16 Years to 65 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
  • Presence of myotonia
  • Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed
  • Body Mass Index (BMI) of \< 35.0 kg/m\^2

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 considered clinically significant by the Investigator
  • Medications specific for the treatment of myotonia within 2 weeks prior to screening
  • Percent predicted forced vital capacity (FVC) \<40%
  • Use of an investigational drug, device, or product within 30 days of 5 half-lives of the study drug (whichever is longer) prior to Screening

Note: Other inclusion and exclusion criteria may apply.

04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
24 participants (estimated)

Study arms

  • Experimental
    PGN-EDODM1

    Participants will be randomized to receive ascending doses of PGN-EDODM1, once every 4 weeks (Q4W) for 12 weeks

    Drug: PGN-EDODM1

  • Placebo comparator
    Placebo

    Participants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks

    Other: Placebo

Interventions

  • DrugPGN-EDODM1

    Administered by intravenous (IV) infusion

  • OtherPlacebo

    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 Day 112

Secondary outcomes

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

    Time frame: Day 1 through Day 84

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

    Time frame: Day 1 through Day 84

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

    Time frame: Day 1 through Day 84

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

    Time frame: Day 1 through Day 84

  5. Change in splicing index in skeletal muscle tissue

    Time frame: Baseline through Day 91

  6. Change in myotonia as measured by video Hand Opening Time (vHOT)

    Time frame: Baseline through Day 112

  7. Hand grip strength

    by dynamometer

    Time frame: Baseline through Day 112

  8. Change in mobility as measured by 10 meter walk/run time

    Time frame: Baseline through Day 112

06

Study locations

12 of 12 sites recruiting
  • University of Calgary
    Calgary, Alberta T2N 4Z6, Canada
    Recruiting
  • Ottawa Hospital Research Institute (OHRI)
    Ottawa, Ontario, Canada
    Recruiting
  • CIUSSS du Saguenay-Lac-Saint-Jean
    Chicoutimi, Quebec, Canada
    Recruiting
  • Montreal Neurological Institute
    Montreal, Quebec H3A 2B4, Canada
    Recruiting
  • Pacific Clinical Research Network Auckland
    Takapuna, Auckland 0622, New Zealand
    Recruiting
  • Pusan National University Yangsan Hospital
    Pusan, Gyeongsangnam-do, South Korea
    Recruiting
  • Kyungpook National University Chilgok Hospital
    Daegu, South Korea
    Recruiting
  • Seoul National University Hospital
    Seoul, South Korea
    Recruiting
  • Salford Royal Hospital
    Salford, England, United Kingdom
    Recruiting
  • University College London Hospitals NHS Foundation Trust
    London, UK, United Kingdom
    Recruiting
  • Cardiff and Vale Hospital
    Cardiff, United Kingdom
    Recruiting
  • Newcastle Upon Tyne Hospitals
    Newcastle upon Tyne, NE7 7DN, United Kingdom
    Recruiting
07

Registry details

Key details

Study ID
NCT06667453
Lead sponsor
PepGen Inc
Responsible party
Sponsor
First posted
Oct 31, 2024
Start date
Dec 10, 2024
Primary completion
Mar 2027 (estimated)
Completion
Mar 2027 (estimated)
Last update
Jul 29, 2026

Study contacts

PepGen
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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