Status:
ACTIVE_NOT_RECRUITING
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
Lead Sponsor:
PepGen Inc
Conditions:
Myotonic Dystrophy 1
Eligibility:
All Genders
18-60 years
Phase:
PHASE1
Brief 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...
Eligibility Criteria
Inclusion
- 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
Exclusion
- Congenital DM1
- Known history or presence of any clinically significant conditions that may interfere with study safety assessments
- Abnormal laboratory tests at screening
- Medications specific for the treatment of myotonia within 2 weeks prior to screening
- Percent predicted forced vital capacity (FVC) \<40%
- Note: Other inclusion and exclusion criteria may apply.
Key Trial Info
Start Date :
December 12 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2025
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT06204809
Start Date
December 12 2023
End Date
October 1 2025
Last Update
October 6 2025
Active Locations (12)
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1
UCI Center for Clinical Research
Irvine, California, United States, 92697
2
Stanford University
Palo Alto, California, United States, 94304
3
Rare Disease Research
Atlanta, Georgia, United States, 30329
4
University of Kansas Medical Center
Fairway, Kansas, United States, 66205