A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment

Duchenne Muscular Dystrophy Clinical Trial

— CONNECT1-EDO51  

Official title:

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06079736
• Other study ID #: PGN-EDO51-102
• Status: Recruiting
• Phase: Phase 2
• Start date: January 8, 2024
• Est. completion date: May 2027

Study information

• Verified date: May 2024
• Source: PepGen Inc
• Contact: Associate Director, Patient Advocacy
• Phone: 781-797-0979
• Email: clinicaltrials[@]pepgen.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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. The study consists of 3 periods: A Screening Period (up to 45 days), a Treatment and Observation Period (16 weeks), and an Extension Period (108 weeks).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: May 2027
• Est. primary completion date: March 2025
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 8 Years and older

Eligibility

Inclusion Criteria:

• Males by birth, age at least 8 years at the time of consent/assent provided
• Confirmed diagnosis of DMD able to be corrected by skipping Exon 51
• Body weight at least 25kg at Screening
• Performance of Upper Limb (PUL) 2.0 entry score of at least 3 at Screening (assessing upper limb function in ambulant and non-ambulant individuals with DMD)

Exclusion Criteria:

• 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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• PGN-EDO51
IV infusion

Locations

Country	Name	City	State
Canada	Stan Cassidy Centre for Rehabilitation	Fredericton	New Brunswick
Canada	Children's Hospital of Eastern Ontario (CHEO)	Ottawa	Ontario
Canada	CHU de Québec	Québec	Quebec
Canada	The Hospital for Sick Children (SickKids)	Toronto	Ontario

Sponsors (1)

Lead Sponsor	Collaborator
PepGen Inc

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period)	Adverse events and serious adverse events	Baseline to Week 16
Primary	Adverse events and serious adverse events (long-term safety and tolerability of PGN-EDO51 in LTE period)	Adverse events and serious adverse events	Baseline to Week 108
Secondary	Plasma pharmacokinetic (PK) parameters (MAD period)	Maximum observed plasma concentration of PGN-EDO51	Baseline to Week 12
Secondary	Plasma pharmacokinetic (PK) parameters (MAD period)	Time to maximum observed plasma concentration of PGN-ED051	Baseline to Week 12
Secondary	Plasma pharmacokinetic (PK) parameters (MAD period)	Apparent terminal half-life of PGN-EDO51	Baseline to Week 12
Secondary	Plasma pharmacokinetic (PK) parameters (MAD period)	Area under the curve for concentration time of PGN-EDO51	Baseline to Week 12
Secondary	PK Plasma levels (LTE period)	PK sampling for PGN-EDO51 and PGN-PMO51 plasma levels	Baseline to Week 104
Secondary	Skeletal muscle concentration (MAD period)	Change from baseline in skeletal muscle concentration of PGN-EDO51 after multiple doses	Baseline to Week 13
Secondary	Dystrophin Levels (MAD period)	Change from baseline in dystrophin levels measured after multiple doses	Baseline to Week 13