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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 is a Phase 1 study that is actively recruiting participants, run by Precision BioSciences. The registered enrollment target is 18 participants.
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07429240
The ClinicalTrials.gov registry entry for NCT07429240 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 18 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Precision BioSciences, which has 5 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which PBGENE-DMD (IV) is the first listed.
NCT07429240 reports 0 study locations.
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.
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