A Phase 2 interventional study of PGN-EDO51 in Duchenne Muscular Dystrophy, sponsored by PepGen Inc. Terminated at 5 sites in Canada. Open to male participants aged 6 Years to 16 Years. Per ClinicalTrials.gov, last updated 2025-12-02.
Sponsored by PepGen Inc · Phase 2, Interventional, and Treatment
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.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 108 are open to participants now.
This study's enrollment of 7 is below the median of 26 across 332 interventional studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →PepGen Inc is the lead sponsor of 5 studies on the registry; 2 are open to participants now.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: PGN-EDO51
Drug: PGN-EDO51
IV infusion
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
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
Time frame: Baseline to Week 16
Dystrophin Levels (MAD period)
Change from baseline in dystrophin levels measured after multiple doses
Time frame: Baseline to Week 16
Ran from Jan 2024 to primary completion in Aug 2025, 19 months.
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This study is terminated, as verified in Dec 2025. You cannot join it, but the record below documents what was studied.
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Muscular Dystrophy, Duchenne →
PepGen Inc