Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants with Myotonic Dystrophy Type 1
Purpose
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).
Condition
- Myotonic Dystrophy 1
Eligibility
- Eligible Ages
- Between 18 Years and 50 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
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 - 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.
Study Design
- Phase
- Phase 1
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental PGN-EDODM1 |
PGN-EDODM1 for infusion |
|
|
Placebo Comparator Placebo |
0.9% NaCl |
|
Recruiting Locations
Massachusetts General Hospital
Boston, Massachusetts 02114
Boston, Massachusetts 02114
Contact:
Thurman Wheeler, MD (PI)
617-726-7506
Thurman Wheeler, MD (PI)
617-726-7506
More Details
- Status
- Recruiting
- Sponsor
- PepGen Inc