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TerminatedNCT06079736CONNECT1-EDO51Updated Dec 2, 2025

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

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

Why this study was terminated
PGN-EDO51 development terminated by Sponsor
Phase
Phase 2
Study type
Interventional
Enrollment
7
Allocation
Non-randomized
Ages
6 Years to 16 Years
Sex
Male
01

Study summary

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.

02

Conditions studied

  • Duchenne Muscular Dystrophy

Keywords

  • Enhanced Delivery Oligonucleotide
  • Peptide-conjugated phosphorodiamidate
  • Morpholino oligomer
  • Oligonucleotide
  • Exon 51
  • Next-generation oligonucleotide
  • Cell-penetrating peptide
  • Muscular Dystrophies
  • Neuromuscular Disease
  • Dystrophin production
  • Splice correcting oligonucleotide
  • Endosomal Escape
  • Delivery to the cell nucleus
  • Antisense oligonucleotide
  • phosphorodiamidate morpholino oligomer (PPMO)
03

Who can participate

Ages eligible
6 Years to 16 Years
Sexes eligible
Male
Accepts healthy volunteers
No

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

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
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
7 participants (actual)

Study arms

  • Experimental
    PGN-EDO51 at Dose Level 1 every 4 weeks

    Drug: PGN-EDO51

  • Experimental
    PGN-EDO51 at Dose Level 2 every 4 weeks

    Drug: PGN-EDO51

Interventions

  • DrugPGN-EDO51

    IV infusion

05

What researchers measure

Primary outcomes

  1. 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

  2. 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

  1. Plasma pharmacokinetic (PK) parameters (MAD period)

    Maximum observed plasma concentration of PGN-EDO51

    Time frame: Baseline to Week 12

  2. Plasma pharmacokinetic (PK) parameters (MAD period)

    Time to maximum observed plasma concentration of PGN-EDO51

    Time frame: Baseline to Week 12

  3. Plasma pharmacokinetic (PK) parameters (MAD period)

    Apparent terminal half-life of PGN-EDO51

    Time frame: Baseline to Week 12

  4. Plasma pharmacokinetic (PK) parameters (MAD period)

    Area under the curve for concentration time of PGN-EDO51

    Time frame: Baseline to Week 12

  5. PK Plasma levels (LTE period)

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

    Time frame: Baseline to Week 104

  6. 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

  7. Dystrophin Levels (MAD period)

    Change from baseline in dystrophin levels measured after multiple doses

    Time frame: Baseline to Week 16

06

Study locations

5 sites
  • British Columbia Children's Hospital
    Vancouver, British Columbia V6H1G9, Canada
  • Stan Cassidy Centre for Rehabilitation
    Fredericton, New Brunswick E3B0C7, Canada
  • Children's Hospital of Eastern Ontario (CHEO)
    Ottawa, Ontario K1H8L1, Canada
  • The Hospital for Sick Children (SickKids)
    Toronto, Ontario M5G0A4, Canada
  • CHU de Québec
    Québec, Quebec G1V4G2, Canada
07

Registry details

Key details

Study ID
NCT06079736
Lead sponsor
PepGen Inc
Responsible party
Sponsor
First posted
Oct 12, 2023
Start date
Jan 3, 2024
Primary completion
Aug 28, 2025
Completion
Aug 28, 2025
Last update
Dec 2, 2025

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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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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