Recruiting
Phase 2

PGN-EDODM1

Sponsor:

PepGen Inc

Code:

NCT06667453

Conditions

Myotonic Dystrophy 1

Eligibility Criteria

Sex: All

Age: 16 - 65

Healthy Volunteers: Not accepted

Interventions

PGN-EDODM1

Placebo

Study Details

Brief 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.

Conditions

Myotonic Dystrophy 1

Study ID

NCT06667453

Start date

Dec 10, 2024

Status verified date

Jul, 2026

Completion date

Mar, 2027

Anticipated

Primary completion date

Mar, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 16 - 65

Healthy Volunteers: Not accepted

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:

  • 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.

Study Design

Enrollment

24 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: PGN-EDODM1

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

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

Interventions

PGN-EDODM1

Administered by intravenous (IV) infusion

Placebo

Administered by intravenous (IV) infusion

Primary outcome measure

  • Safety and tolerability as assessed by number of participants with Adverse Events (AEs) [ Time Frame: Baseline through Day 112 ]

Central Contacts and Locations

Central contacts

PepGen

781-797-0979clinicaltrials@pepgen.com

Locations

University of Calgary

Recruiting

Calgary, Alberta, Canada, T2N 4Z6

Contacts

Patient Advocacy

781-797-0979clinicaltrials@pepgen.com

Ottawa Hospital Research Institute (OHRI)

Recruiting

Ottawa, Ontario, Canada

Contacts

Jessica MacGregor

613-798-5555jemacgregor@ohri.ca

CIUSSS du Saguenay-Lac-Saint-Jean

Recruiting

Chicoutimi, Quebec, Canada

Contacts

Jessica Tremblay

418-541-1000jessica.tremblay.agt@ssss.gouv.qc.ca

Montreal Neurological Institute

Recruiting

Montreal, Quebec, Canada, H3A 2B4

Contacts

Patient Advocacy

781-797-0979clinicaltrials@pepgen.com

More Information

Sponsor

PepGen Inc

Last update posted

Jul 29, 2026

Last verified

Jul, 2026

Keywords

  • DM1
  • Myotonic Dystrophy 1
  • Myotonic Dystrophy
  • PepGen
  • PGN-EDODM1
  • Myotonic Muscular Dystrophy
  • Steinert&#39;s Disease

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by PepGen Inc on 2026-07-29.