Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
NCT ID: NCT06204809
Last Updated: 2025-10-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1
24 participants
INTERVENTIONAL
2023-12-12
2025-10-31
Brief Summary
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The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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PGN-EDODM1
PGN-EDODM1 for infusion
PGN-EDODM1 for infusion
Single dose of PGN-EDODM1 by intravenous (IV) infusion
Placebo
0.9% NaCl
Placebo
Administered by IV infusion
Interventions
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PGN-EDODM1 for infusion
Single dose of PGN-EDODM1 by intravenous (IV) infusion
Placebo
Administered by IV infusion
Eligibility Criteria
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Inclusion Criteria
* 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
* 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%
18 Years
60 Years
ALL
No
Sponsors
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PepGen Inc
INDUSTRY
Responsible Party
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Locations
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UCI Center for Clinical Research
Irvine, California, United States
Stanford University
Palo Alto, California, United States
Rare Disease Research
Atlanta, Georgia, United States
University of Kansas Medical Center
Fairway, Kansas, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
University of Rochester Medical Center
Rochester, New York, United States
Virginia Commonwealth University
Richmond, Virginia, United States
University of Calgary
Calgary, Alberta, Canada
Ottawa Hospital Research Institute (OHRI)
Ottawa, Ontario, Canada
CIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi, Quebec, Canada
University College London Hospital
London, UK, United Kingdom
Salford Royal Hospital
Salford, , United Kingdom
Countries
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Other Identifiers
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PGN-EDODM1-101
Identifier Type: -
Identifier Source: org_study_id
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