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A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT2-EDO51)

Trial Profile

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT2-EDO51)

Status: Withdrawn prior to enrolment
Phase of Trial: Phase II

Latest Information Update: 30 Jun 2025

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At a glance

  • Drugs PGN-EDO51 (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions; Registrational; Therapeutic Use
  • Acronyms CONNECT2-EDO51
  • Sponsors PEPGENE

Most Recent Events

  • 24 Jun 2025 Status changed from suspended to withdrawn prior to enrolment because Global PGN-EDO51 development voluntarily discontinued by Sponsor.
  • 08 May 2025 According to a Pepgen Corporation media release, dystrophin production data from 10 mg/kg cohort is expected in Q3 2025.
  • 18 Mar 2025 Planned End Date changed from 30 Apr 2029 to 30 Apr 2030.

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