A Phase 2 interventional study of IV infusion and Placebo in Duchenne Muscular Dystrophy (DMD), sponsored by PepGen Inc. Withdrawn. Open to male participants aged 6 Years to 16 Years. Per ClinicalTrials.gov, last updated 2025-06-24.
Sponsored by PepGen Inc · Phase 2, Interventional, and Treatment
The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
The primary purpose of the MAD period is to evaluate the safety and tolerability and levels of dystrophin after multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). During the MAD period, participants will be randomized to either receive PGN-EDO51 or placebo in a 3:1 fashion, meaning that participants have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during this period. The primary purpose of the open-label LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period. All participants who roll-over into the LTE will receive PGN-EDO51 (no placebo in the LTE).
Exclusion Criteria:
Drug: IV infusion · Other: Placebo
Drug: IV infusion · Other: Placebo
IV infusion
IV infusion
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the MAD period)
Time frame: Signing of informed consent to Week 28
Dystrophin levels (MAD period)
Time frame: Baseline to Week 28
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the LTE period)
Time frame: Signing of informed consent to Week 108
Plasma pharmacokinetic (PK) parameters (MAD period)
Maximum observed plasma concentration of PGN-EDO51
Time frame: Baseline to Week 28
Plasma pharmacokinetic (PK) parameters (MAD period)
Time to maximum observed plasma concentration of PGN-EDO51
Time frame: Baseline to Week 28
Plasma pharmacokinetic (PK) parameters (MAD period)
Apparent terminal half-life of PGN-EDO51
Time frame: Baseline to Week 28
Plasma pharmacokinetic (PK) parameters (MAD period)
Area under the curve for concentration time of PGN-EDO51
Time frame: Baseline to Week 28
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 28
Plasma pharmacokinetic (PK) parameters (LTE period)
PK sampling for PGN-EDO51 and PGN-PMO51 plasma levels
Time frame: Baseline to Week 104
No study locations are listed for this record.
This study is withdrawn, as verified in Jun 2025. You cannot join it, but the record below documents what was studied.
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PepGen Inc