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NCT06833931: CONNECT2-EDO51
A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
Phase 2 trial testing IV infusion in Duchenne Muscular Dystrophy (DMD). Withdrawn.
28 May 2025
Quick facts
| Lead sponsor | PepGen Inc |
|---|---|
| Phase | Phase 2 |
| Status | Withdrawn |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | sequential |
| Masking | quadruple |
| Primary purpose | treatment |
| Start date | 17 December 2024 |
| Primary completion | 28 May 2025 |
| Estimated completion | 28 May 2025 |
Drugs / interventions tested
- IV infusion
- Placebo
Conditions studied
- Duchenne Muscular Dystrophy (DMD) — all drugs for Duchenne Muscular Dystrophy (DMD) →
Sponsor
PepGen Inc — full company profile →
Who can join
Adults 6 to 16, male only, with Duchenne Muscular Dystrophy (DMD). Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The 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).
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
RNA Therapeutics for Duchenne Muscular Dystrophy: Exon Skipping, RNA Editing, and Translational Insights from Genome-Edited Microminipig Models.
Chassin A, Ono H, Ashida Y, Imamura M, et al · · 2026 · PMID 41898615 · DOI 10.3390/ijms27062755
Verify or expand the search:
- PubMed search for NCT06833931
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
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Trials testing the same drug.
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Other recruiting trials for Duchenne Muscular Dystrophy (DMD)
Currently open trials in the same condition.
- NCT07038824 — A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficac · Phase 1, PHASE2 · recruiting
- NCT07129954 — Fear of Falling in Muscular Dystrophy · NA · recruiting
- NCT07037862 — A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficac · Phase 1, PHASE2 · recruiting
- NCT06755138 — Research on the Relationship Between Scoliosis, Pain, Quality of Life, and Trunk Muscle Compensation Patterns Among Pati · recruiting
- NCT06773988 — Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy · recruiting
Other PepGen Inc trials
Trials by the same sponsor.
- NCT06079736 — A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment · Phase 2 · terminated
- NCT06204809 — Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 · Phase 1 · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT06833931 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by PepGen Inc
- Last refreshed: 24 June 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06833931.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing