NCT06833931

Brief Summary

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).

Trial Health

15
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Timeline
Completed

Started Dec 2024

Shorter than P25 for phase_2

Status
withdrawn

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

Study Start

First participant enrolled

December 17, 2024

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

February 13, 2025

Completed
6 days until next milestone

First Posted

Study publicly available on registry

February 19, 2025

Completed
3 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 28, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 28, 2025

Completed
Last Updated

June 24, 2025

Status Verified

June 1, 2025

Enrollment Period

5 months

First QC Date

February 13, 2025

Last Update Submit

June 18, 2025

Conditions

Duchenne Muscular Dystrophy (DMD)

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 (3)

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

    Signing of informed consent to Week 28

  • Dystrophin levels (MAD period)

    Baseline to Week 28

  • Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the LTE period)

    Signing of informed consent to Week 108

Secondary Outcomes (6)

  • Plasma pharmacokinetic (PK) parameters (MAD period)

    Baseline to Week 28

  • Plasma pharmacokinetic (PK) parameters (MAD period)

    Baseline to Week 28

  • Plasma pharmacokinetic (PK) parameters (MAD period)

    Baseline to Week 28

  • Plasma pharmacokinetic (PK) parameters (MAD period)

    Baseline to Week 28

  • Skeletal muscle concentration of PGN-EDO51 (MAD period)

    Baseline to Week 28

  • +1 more secondary outcomes

Study Arms (2)

PGN-EDO51 at Dose Level 1 or Placebo every 4 weeks

EXPERIMENTAL
Drug: IV infusionOther: Placebo

PGN-EDO51 at Dose Level 2 or Placebo every 4 weeks

EXPERIMENTAL
Drug: IV infusionOther: Placebo

Interventions

IV infusionDRUG

IV infusion

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

IV infusion

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

Eligibility Criteria

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

You may qualify if:

  • Confirmed diagnosis of DMD with a genetic alteration that can be treated by skipping exon 51
  • Body weight at least 25kg (55lbs)
  • Performance of Upper Limb (PUL) 2.0 entry score of at least 4

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

MeSH Terms

Conditions

Muscular Dystrophy, DuchenneMuscular DystrophiesNeuromuscular Diseases

Interventions

Infusions, Intravenous

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Administration, IntravenousDrug Administration RoutesDrug TherapyTherapeuticsInfusions, Parenteral
0

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 13, 2025

First Posted

February 19, 2025

Study Start

December 17, 2024

Primary Completion

May 28, 2025

Study Completion

May 28, 2025

Last Updated

June 24, 2025

Record last verified: 2025-06