PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy
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 potenti
| Condition(s) | Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD |
|---|---|
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Study type | Interventional |
| 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. |
| Who can participate | Inclusion Criteria: 1. Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent 2. Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 \[inclusive\]) 3. Clinical phenotype consistent with DMD in the opinion of the Investigator 4. Ability to complete age-appropriate motor testing assessments requirements. Participants aged 2 to \< 4 years at the time of screening must: 1. Be able to walk at least 10 meters independently (without assistive devices). 2. Be able to rise from the floor without physical assistance (use of a Gowers' maneuver is acceptable). Participants aged 4 to 7 years at the time of screening must: 3. Be able to walk at least 100 meters independently (without assistive devices). 4. Have an NSAA total score between 16 and |
| Ages | 2 Years to 7 Years |
| Sex | Male |
| Lead sponsor | Precision BioSciences, Inc. |
| Locations | Little Rock, Arkansas, United States |
| Start date | 2026-04-24 |
| NCT ID | NCT07429240 |
| Official listing | https://clinicaltrials.gov/study/NCT07429240 |