A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)
Status: Active_not_recruiting
Location: See all (12) locations...
Intervention Type: Drug, Other
Study Type: Interventional
Study Phase: Phase 1
SUMMARY
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).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
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
Locations
United States
California
UCI Center for Clinical Research
Irvine
Stanford University
Palo Alto
Georgia
Rare Disease Research
Atlanta
Kansas
University of Kansas Medical Center
Fairway
Massachusetts
Massachusetts General Hospital
Boston
New York
University of Rochester Medical Center
Rochester
Virginia
Virginia Commonwealth University
Richmond
Other Locations
Canada
University of Calgary
Calgary
CIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi
Ottawa Hospital Research Institute (OHRI)
Ottawa
United Kingdom
University College London Hospital
London
Salford Royal Hospital
Salford
Time Frame
Start Date: 2023-12-12
Completion Date: 2025-10
Participants
Target number of participants: 24
Treatments
Experimental: PGN-EDODM1
PGN-EDODM1 for infusion
Placebo_comparator: Placebo
0.9% NaCl
Related Therapeutic Areas
Sponsors
Leads: PepGen Inc