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
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.
126 studies on the registry are indexed under Myotonic Dystrophy; 50 are open to participants now.
This study's planned enrollment of 24 is below the median of 40 across 67 interventional studies indexed under Myotonic Dystrophy.
Browse Myotonic Dystrophy studies →PepGen Inc is the lead sponsor of 5 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Note: Other inclusion and exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will be randomized to receive ascending doses of PGN-EDODM1, once every 4 weeks (Q4W) for 12 weeks
Drug: PGN-EDODM1
Participants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks
Other: Placebo
Administered by intravenous (IV) infusion
Administered by intravenous (IV) infusion
Safety and tolerability as assessed by number of participants with Adverse Events (AEs)
Time frame: Baseline through Day 112
Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
Time frame: Day 1 through Day 84
Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1
Time frame: Day 1 through Day 84
Apparent Terminal Half-Life (t½) of PGN-EDODM1
Time frame: Day 1 through Day 84
Area Under the Concentration-time Curve of PGN-EDODM1
Time frame: Day 1 through Day 84
Change in splicing index in skeletal muscle tissue
Time frame: Baseline through Day 91
Change in myotonia as measured by video Hand Opening Time (vHOT)
Time frame: Baseline through Day 112
Hand grip strength
by dynamometer
Time frame: Baseline through Day 112
Change in mobility as measured by 10 meter walk/run time
Time frame: Baseline through Day 112
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