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IV infusion · CONNECT2-EDO51

A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Ensayo de PepGen Inc. en Distrofia muscular de Duchenne.

Más ensayos de: Distrofia muscular de Duchenne.

Fase

Fase 2

Estado

Retirado antes de empezar

Fin del objetivo primario

may 2025

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, cuádruple ciego. Comenzó en dic 2024.

Este ensayo se detuvo. Motivo declarado por el promotor (del registro, en inglés): «Global PGN-EDO51 development voluntarily discontinued by Sponsor».

Qué mide

Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the MAD period) (Signing of informed consent to Week 28) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThe study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks). The primary purpose of the MAD period is to evaluate the safety and tolerability and levels of dystrophin after multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). During the MAD period, participants will be randomized to either receive PGN-EDO51 or placebo in a 3:1 fashion, meaning that participants have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during this period. The primary purpose of the open-label LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period. All participants who roll-over into the LTE will receive PGN-EDO51 (no placebo in the LTE).

Más ensayos de PEPG

Todos los de PEPG, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT06833931), actualizada en jun 2025.