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

Recruiting
18-50 years
Both
Phase 1

Myotonic Dystrophy Type 1 (DM1) is a genetic muscle disorder that causes muscles to be weak and stiff. This study is testing a new medicine called PGN-EDODM1 to see if it's safe for people with DM1. The medicine is given through a needle in a vein, called an intravenous (IV) dose. The study has two parts: a Screening Period lasting up to 30 days to check if you can join, and a Treatment and Observation Period lasting 16 weeks to see how the medicine works.

To join, you need to have a confirmed diagnosis of DM1 with specific genetic markers, and your muscles should not be very weak. You cannot join if you have other health problems that might make the study unsafe for you, or if you're taking certain medications.

  • The study lasts for about 4 months.
  • You will receive a single IV dose of the study medication.
  • There are specific health requirements to join the study.

Make sure to talk to your doctor to see if this study is right for you. 🧬💉

Study details
    Myotonic Dystrophy 1

NCT06204809

PepGen Inc

24 January 2024

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