RecruitingPhase 2

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

Canada, New Zealand, United Kingdom24 participantsStarted 2024-12-10

Plain-language 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.

Who can participate

Age range

16 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Safety and tolerability as assessed by number of participants with Adverse Events (AEs)

Timeframe: Baseline through Day 112

Trial details

NCT IDNCT06667453

SponsorPepGen Inc

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-03

Contact for this trial

PepGen

781-797-0979 clinicaltrials@pepgen.com

View on ClinicalTrials.gov More Myotonic Dystrophy 1 trials

Who can participate

Age range

16 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.