Actively Recruiting
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Led by PepGen Inc · Updated on 2026-04-28
24
Participants Needed
8
Research Sites
115 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of myotonic dystrophy type 1 with at least 100 CTG repeats in the DMPK gene
- Presence of muscle stiffness (myotonia)
- Sufficient muscle mass in both tibialis anterior muscles to safely perform a needle biopsy
- Body mass index (BMI) below 35.0 kg/m²
You will not qualify if you...
- Congenital myotonic dystrophy type 1
- Any clinically significant condition affecting study safety assessments
- Clinically significant abnormal lab tests at screening
- Use of medications specifically for myotonia within 2 weeks prior to screening
- Forced vital capacity (FVC) under 40%
- Use of any investigational drug, device, or product within 30 days or 5 half-lives of the study drug before screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 8 locations
1
University of Calgary
Calgary, Alberta, Canada, T2N 4Z6
Actively Recruiting
2
Ottawa Hospital Research Institute (OHRI)
Ottawa, Ontario, Canada
Actively Recruiting
3
CIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi, Quebec, Canada
Actively Recruiting
4
Montreal Neurological Institute
Montreal, Quebec, Canada, H3A 2B4
Actively Recruiting
5
Pacific Clinical Research Network Auckland
Takapuna, Auckland, New Zealand, 0622
Active, Not Recruiting
6
Salford Royal Hospital
Salford, England, United Kingdom
Actively Recruiting
7
University College London Hospitals NHS Foundation Trust
London, UK, United Kingdom
Actively Recruiting
8
Newcastle Upon Tyne Hospitals
Newcastle upon Tyne, United Kingdom, NE7 7DN
Actively Recruiting
Research Team
P
PepGen
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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