NCT06079736TerminatedPhase 2
A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
A Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)
Source: ClinicalTrials.gov · Last updated 2 Dec 2025
Reason given: PGN-EDO51 development terminated by Sponsor
Brief summary (registry, in English)
The study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks). The primary purpose of the MAD period is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne Muscular Dystrophy (DMD). The primary purpose of the LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period.
- Status (registry)
- Terminated
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 7 (actual)
- First submitted
- 29 Sept 2023
- Start date
- 3 Jan 2024
- Primary completion date
- 28 Aug 2025
- Lead sponsor
- PepGen Inc
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT06079736
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Treatment with any gene replacement therapy for the treatment of DMD at any time
- Current or recent systemic infection within 2 weeks prior to Screening or infection requiring IV antibiotics within 4 weeks prior to Screening
- Recent surgery requiring anesthesia within 3 months prior to Screening or expected surgery requiring general anesthesia during the study
Other illness
- Known history or presence of any clinically significant conditions that may interfere with study safety assessments
Who the study is looking for
- Confirmed diagnosis of DMD able to be corrected by skipping Exon 51
- Body weight at least 18kg at Screening
- Performance of Upper Limb (PUL) 2.0 entry score of at least 4 at Screening (assessing upper limb function in ambulant and non-ambulant individuals with DMD)
Age in the registry: 6 years to 16 years · Sex: Male · Does not accept healthy volunteers
Sites (5)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Stan Cassidy Centre for Rehabilitation | Fredericton | Canada | Unknown status |
| Children's Hospital of Eastern Ontario (CHEO) | Ottawa | Canada | Unknown status |
| CHU de Quebec | Québec | Canada | Unknown status |
| The Hospital for Sick Children (SickKids) | Toronto | Canada | Unknown status |
| British Columbia Children's Hospital | Vancouver | Canada | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.