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PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)

NCT07429240 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2026-06-23

No results posted yet for this 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.

Conditions

  • Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD

Interventions

BIOLOGICAL

PBGENE-DMD (IV)

Participants will receive a single dose of PBGENE-DMD

Sponsors & Collaborators

  • Precision BioSciences, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
7 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-04-24
Primary Completion
2029-11-30
Completion
2029-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07429240 on ClinicalTrials.gov