A Phase 1 interventional study of PGN-EDODM1 for infusion and Placebo in Myotonic Dystrophy 1, sponsored by PepGen Inc. Completed at 12 sites in 3 countries. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2026-02-13.
Sponsored by PepGen Inc · Phase 1, Interventional, and Treatment
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).
The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).
126 studies on the registry are indexed under Myotonic Dystrophy; 50 are open to participants now.
This study's 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.
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.
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.
PGN-EDODM1 for infusion
Drug: PGN-EDODM1 for infusion
0.9% NaCl
Other: Placebo
Single dose of PGN-EDODM1 by intravenous (IV) infusion
Administered by IV infusion
Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs
Time frame: Baseline to Week 16
Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
Time frame: Baseline up to Day 3
Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1
Time frame: Baseline up to Day 3
Apparent Terminal Half-Life (t½) of PGN-EDODM1
Time frame: Baseline up to Day 3
Area Under the Concentration-time Curve of PGN-EDODM1
Time frame: Baseline up to Day 3
This study is completed, as verified in Feb 2026. You cannot join it, but the record below documents what was studied.
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PepGen Inc