Trial Profile

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)

Status: Completed

Phase of Trial: Phase I

Latest Information Update: 16 Feb 2026

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At a glance

13 Feb 2026 Status changed from active, no longer recruiting to completed.
12 Nov 2025 According to a Pepgen Inc media release, the company reported topline results from the 15 mg/kg cohort of PGN-EDODM1.
12 Nov 2025 According to a Pepgen Inc media release, In October 2025, PepGen gave an oral presentation at the Oligonucleotide Therapeutics Society (OTS) Annual Meeting which included a review of the FREEDOM clinical data.

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