Status:
TERMINATED
A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
Lead Sponsor:
PepGen Inc
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
6-16 years
Phase:
PHASE2
Brief Summary
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 ...
Eligibility Criteria
Inclusion
- 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)
Exclusion
- Known history or presence of any clinically significant conditions that may interfere with study safety assessments
- 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
Key Trial Info
Start Date :
January 3 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 28 2025
Estimated Enrollment :
7 Patients enrolled
Trial Details
Trial ID
NCT06079736
Start Date
January 3 2024
End Date
August 28 2025
Last Update
December 2 2025
Active Locations (5)
Enter a location and click search to find clinical trials sorted by distance.
1
British Columbia Children's Hospital
Vancouver, British Columbia, Canada, V6H1G9
2
Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick, Canada, E3B0C7
3
Children's Hospital of Eastern Ontario (CHEO)
Ottawa, Ontario, Canada, K1H8L1
4
The Hospital for Sick Children (SickKids)
Toronto, Ontario, Canada, M5G0A4