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Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

Study on PGN-EDODM1 safety in Myotonic Dystrophy Type 1 patients.

Recruiting
18-60 years
All
Phase 1

This study looks at the safety of a drug called PGN-EDODM1 for people with Myotonic Dystrophy Type 1 (DM1), a genetic disorder that causes muscle problems. Participants will get a single dose through an IV (a tube into a vein). The study lasts about 4 months, with two parts: a Screening Period (up to 30 days) to check if you can join, and a Treatment and Observation Period (16 weeks) to monitor the effects.

To join, you need a confirmed DM1 diagnosis and should be able to move certain muscles well. You can't join if you have a type of DM1 present at birth, other major health issues, or if you're taking certain medicines.

  • The study takes about 4 months in total.
  • Participants will get one IV dose and be monitored.
  • Certain health conditions or medications may disqualify you from joining.
Study details
    Myotonic Dystrophy 1

NCT06204809

PepGen Inc

14 June 2025

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A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

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