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NCT07429240 · ClinicalTrials.gov registry record · Phase 1
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
A Phase 1 study, sponsored by Precision BioSciences.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 18
- Enrollment target
NCT07429240: Recruiting Phase 1 study, sponsored by Precision BioSciences.
NCT07429240 is a Phase 1 study that is actively recruiting participants, run by Precision BioSciences. The registered enrollment target is 18 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (70% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07429240, a Phase 1 study, is actively recruiting participants, sponsored by Precision BioSciences.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 18 participants
- Enrollment target
Study Summary
The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.
Primary Outcome
Adverse events and serious adverse events that occur or worsen after initiation of the investigational treatment
Interventions
- BIOLOGICAL PBGENE-DMD (IV)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 18 participants |
| Start Date | 2026-04-24 |
| Est. Completion | 2029-12 |
| Phase | Phase 1 |
What NCT07429240 shows while recruiting
NCT07429240 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 18 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (70% lower).
The record links to 0 conditions, and to 1 intervention - of which PBGENE-DMD (IV) is the first listed.
NCT07429240 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT07429240 about?
NCT07429240 is a clinical study titled "PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)". The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative appro...
What is the current status of trial NCT07429240?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 18 participants. The study started on 2026-04-24. Estimated completion is 2029-12.
What interventions are being tested in trial NCT07429240?
The interventions under investigation include: PBGENE-DMD (IV) (BIOLOGICAL).
Who is sponsoring clinical trial NCT07429240?
This trial is sponsored by Precision BioSciences, which has 5 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT07429240's enrollment target sits among peer trials
18 1720th of 2000 higher than 216 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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