NCT06079736

Brief 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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
7

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2024

Geographic Reach
1 country

5 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 29, 2023

Completed
13 days until next milestone

First Posted

Study publicly available on registry

October 12, 2023

Completed
3 months until next milestone

Study Start

First participant enrolled

January 3, 2024

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 28, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 28, 2025

Completed
Last Updated

December 2, 2025

Status Verified

December 1, 2025

Enrollment Period

1.7 years

First QC Date

September 29, 2023

Last Update Submit

December 1, 2025

Conditions

Duchenne Muscular Dystrophy

Keywords

Enhanced Delivery OligonucleotidePeptide-conjugated phosphorodiamidateMorpholino oligomerOligonucleotideExon 51Next-generation oligonucleotideCell-penetrating peptideMuscular DystrophiesNeuromuscular DiseaseDystrophin productionSplice correcting oligonucleotideEndosomal EscapeDelivery to the cell nucleusAntisense oligonucleotidephosphorodiamidate morpholino oligomer (PPMO)

Outcome Measures

Primary Outcomes (2)

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

    Adverse events and serious adverse events

    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

    Baseline to Week 108

Secondary Outcomes (7)

  • Plasma pharmacokinetic (PK) parameters (MAD period)

    Baseline to Week 12

  • Plasma pharmacokinetic (PK) parameters (MAD period)

    Baseline to Week 12

  • Plasma pharmacokinetic (PK) parameters (MAD period)

    Baseline to Week 12

  • Plasma pharmacokinetic (PK) parameters (MAD period)

    Baseline to Week 12

  • PK Plasma levels (LTE period)

    Baseline to Week 104

  • +2 more secondary outcomes

Study Arms (2)

PGN-EDO51 at Dose Level 1 every 4 weeks

EXPERIMENTAL
Drug: PGN-EDO51

PGN-EDO51 at Dose Level 2 every 4 weeks

EXPERIMENTAL
Drug: PGN-EDO51

Interventions

PGN-EDO51DRUG

IV infusion

PGN-EDO51 at Dose Level 1 every 4 weeksPGN-EDO51 at Dose Level 2 every 4 weeks

Eligibility Criteria

Age6 Years - 16 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

British Columbia Children's Hospital

Vancouver, British Columbia, V6H1G9, Canada

Location

Stan Cassidy Centre for Rehabilitation

Fredericton, New Brunswick, E3B0C7, Canada

Location

Children's Hospital of Eastern Ontario (CHEO)

Ottawa, Ontario, K1H8L1, Canada

Location

The Hospital for Sick Children (SickKids)

Toronto, Ontario, M5G0A4, Canada

Location

CHU de Québec

Québec, Quebec, G1V4G2, Canada

Location

MeSH Terms

Conditions

Muscular Dystrophy, DuchenneMuscular DystrophiesNeuromuscular Diseases

Condition Hierarchy (Ancestors)

Muscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 29, 2023

First Posted

October 12, 2023

Study Start

January 3, 2024

Primary Completion

August 28, 2025

Study Completion

August 28, 2025

Last Updated

December 2, 2025

Record last verified: 2025-12

Locations

CA(5)