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Taking peoplePhase 2

Testing PGN-EDODM1 against placebo in myotonic dystrophy type 1

Tests the safety of repeated PGN-EDODM1 doses in adults with myotonic dystrophy type 1.

Key facts

Who runs it
PepGen Inc
Where
Canada, New Zealand, South Korea, and United Kingdom12 sites in 4 countriesChoose a country to see its sites
Trial code
Registry title
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Status
Recruiting
Phase
Phase 2
People taking part
24 people, planned
Started
10 Dec 2024
Expected to end
Mar 2027
Type of study
Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • PGN-EDODM1
  • Placebo
Record updated
29 July 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for people aged 16 to 65 with myotonic dystrophy type 1 (DM1). It tests how safe repeated doses of PGN-EDODM1 are, compared with placebo.

Age
16 Years to 65 Years
Sex
All sexes
Disease stage
Confirmed DM1 with myotonia (muscles slow to relax after use). Enough muscle in both lower leg (tibialis anterior) muscles for a safe needle biopsy. Body Mass Index (BMI) below 35.0 kg/m^2.
Earlier treatment
No medicines for myotonia within 2 weeks before screening. No other study drug, device or product within 30 days or 5 half-lives of the study drug before screening, whichever is longer.
Biomarker or test result
At least 100 CTG repeats in the DMPK gene.

Main reasons for not taking part

  • DM1 present from birth (congenital DM1)
  • Serious health conditions that could affect the safety checks
  • Abnormal lab tests at screening that the doctor finds important
  • Lung function (predicted FVC) below 40%
  • Recent use of myotonia medicines or other study drugs

The registry criteria, word for word

Show all 10 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • Medications specific for the treatment of myotonia within 2 weeks prior to screening
  • 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
  • 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
  • Congenital DM1
  • Percent predicted forced vital capacity (FVC) <40%

Check if you might fit

Answer for yourself. This is not a decision: only the study team can say whether you can take part.

  • Are you between 16 and 65 years old?

This trial has no sites in United States.

It has sites in 4 countries. Choose a country to see its sites.

About the trial

What the trial wants to find out

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.

The 2 groups in the trial

  • PGN-EDODM1Treatment being tested

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

  • PlaceboPlacebo, for comparison

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

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
Plain-language title
Testing PGN-EDODM1 against placebo in myotonic dystrophy type 1Generated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 15 November 2026.
Fit questions
Built from NCT06667453, who can take part
Record updated
29 July 2026