A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
Status: Recruiting
Location: See all (6) locations...
Intervention Type: Drug, Other
Study Type: Interventional
Study Phase: Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 16
Maximum Age: 60
Healthy Volunteers: f
View:
• 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
Locations
Other Locations
Canada
University of Calgary
RECRUITING
Calgary
CIUSSS du Saguenay-Lac-Saint-Jean
RECRUITING
Chicoutimi
Montreal Neurological Institute
RECRUITING
Montreal
Ottawa Hospital Research Institute (OHRI)
RECRUITING
Ottawa
United Kingdom
University College London Hospitals NHS Foundation Trust
RECRUITING
London
Newcastle Upon Tyne Hospitals
RECRUITING
Newcastle Upon Tyne
Contact Information
Primary
PepGen
clinicaltrials@pepgen.com
781-797-0979
Time Frame
Start Date: 2024-12-10
Estimated Completion Date: 2026-08
Participants
Target number of participants: 24
Treatments
Experimental: PGN-EDODM1
Participants will be randomized to receive ascending doses of PGN-EDODM1, once every 4 weeks (Q4W) for 12 weeks
Placebo_comparator: Placebo
Participants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks
Related Therapeutic Areas
Sponsors
Leads: PepGen Inc