A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)

PepGen

Status and phase

Active, not recruiting

Phase 2

Conditions

Duchenne Muscular Dystrophy

Treatments

Drug: PGN-EDO51

Study type

Interventional

Funder types

Industry

Identifiers

NCT06079736

PGN-EDO51-102

Details and patient eligibility

About

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.

Enrollment

7 patients

Sex

Male

Ages

6 to 16 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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 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