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CompletedPhase 1

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

Key facts

Who runs it
PepGen Inc
Where
Atlanta, Boston, Fairway, Irvine, Palo Alto, Richmond, and Rochester, United StatesSee the 7 sitesAlso in 2 other countries, 5 sites
Trial code
Status
Completed
Phase
Phase 1
People taking part
24 people enrolled
Started
12 Dec 2023
Ended
28 Oct 2025
Type of study
Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • PGN-EDODM1 for infusion
  • Placebo
Record updated
13 February 2026

Who can take part

Age
18 years to 60 years
Sex
Women and men
Healthy volunteers
Cannot take part

The registry criteria, word for word

Show all 8 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
  • 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

Who cannot take part

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

Where in United States

Also in 2 other countries, 5 sites

  • Rare Disease ResearchAtlanta, United States
  • Massachusetts General HospitalBoston, United States
  • University of Kansas Medical CenterFairway, United States
  • UCI Center for Clinical ResearchIrvine, United States
  • Stanford UniversityPalo Alto, United States
  • Virginia Commonwealth UniversityRichmond, United States
  • University of Rochester Medical CenterRochester, United States

About the trial

What the trial wants to find out

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).

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The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).

The 2 groups in the trial

  • PGN-EDODM1Treatment being tested

    PGN-EDODM1 for infusion

    Treatment: PGN-EDODM1 for infusion

  • PlaceboPlacebo, for comparison

    0.9% NaCl

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
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)
Record updated
13 February 2026