FREEDOM-DM1

• Research type

  Research Study

• Full title

  A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1 (FREEDOM-DM1)

• IRAS ID

  1008589

• Contact name

  Hayley Parker

• Contact email

  clinreg@pepgen.com

• Sponsor organisation

  PepGen Inc.

• Research summary

  The purpose of this study is to learn about the effects of an “investigational” medicine, PGN-EDODM1, and to see how safe and tolerable PGN-EDODM1 is for people with myotonic dystrophy type 1 (DM1) compared to placebo.

• REC name

  London - City & East Research Ethics Committee

• REC reference

  23/LO/0854

• Date of REC Opinion

  20 Nov 2023

• REC opinion

  Further Information Favourable Opinion