Status:
RECRUITING
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Lead Sponsor:
PepGen Inc
Conditions:
Myotonic Dystrophy 1
Eligibility:
All Genders
16-60 years
Phase:
PHASE2
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 dy...
Eligibility Criteria
Inclusion Criteria:
- Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
- Medical Research Council (MRC) score of ≥ Grade 4- in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
- Presence of myotonia
- Body Mass Index (BMI) of < 32.0 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.
Key Trial Info
Start Date :
December 10 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 1 2026
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT06667453
Start Date
December 10 2024
End Date
August 1 2026
Last Update
March 2 2026
Active Locations (8)
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1
University of Calgary
Calgary, Alberta, Canada, T2N 4Z6
2
Ottawa Hospital Research Institute (OHRI)
Ottawa, Ontario, Canada
3
CIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi, Quebec, Canada
4
Montreal Neurological Institute
Montreal, Quebec, Canada, H3A 2B4