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

Phase 2TerminatedNCT06079736

PepGen Inc7 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

PGN-EDO51DRUG

IV infusion

Eligibility

Sex: MALEMin age: 6 YearsMax age: 16 Years

Medical Language ↔ Plain English

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

Locations (5)

British Columbia Children's Hospital

Vancouver, British Columbia, Canada

Stan Cassidy Centre for Rehabilitation

Fredericton, New Brunswick, Canada

Children's Hospital of Eastern Ontario (CHEO)

Ottawa, Ontario, Canada

The Hospital for Sick Children (SickKids)

Toronto, Ontario, Canada

Outcomes

Primary Outcomes

Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period)

Adverse events and serious adverse events

Time frame: Baseline to Week 16

Adverse events and serious adverse events (long-term safety and tolerability of PGN-EDO51 in LTE period)

Adverse events and serious adverse events

Time frame: Baseline to Week 108

Secondary Outcomes

Plasma pharmacokinetic (PK) parameters (MAD period)

Maximum observed plasma concentration of PGN-EDO51

Time frame: Baseline to Week 12

Plasma pharmacokinetic (PK) parameters (MAD period)

Time to maximum observed plasma concentration of PGN-EDO51

Time frame: Baseline to Week 12

Plasma pharmacokinetic (PK) parameters (MAD period)

Apparent terminal half-life of PGN-EDO51

Time frame: Baseline to Week 12

Plasma pharmacokinetic (PK) parameters (MAD period)

Area under the curve for concentration time of PGN-EDO51

Time frame: Baseline to Week 12

PK Plasma levels (LTE period)

PK sampling for PGN-EDO51 and PGN-PMO51 plasma levels

Time frame: Baseline to Week 104

Skeletal muscle concentration of PGN-EDO51 (MAD period)

Change from baseline in skeletal muscle concentration of PGN-EDO51 after multiple doses

Data from ClinicalTrials.gov

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