PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
A Phase 1/2a, Multi-center, Open-label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of PBGENE-DMD in Participants With Duchenne Muscular Dystrophy (FUNCTION-DMD)
Precision BioSciences, Inc.
18 participants
Apr 24, 2026
INTERVENTIONAL
Conditions
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.
Eligibility
Inclusion Criteria13
- Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent
- Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 \[inclusive\])
- Clinical phenotype consistent with DMD in the opinion of the Investigator
- Ability to complete age-appropriate motor testing assessments requirements.
- Participants aged 2 to < 4 years at the time of screening must:
- Be able to walk at least 10 meters independently (without assistive devices).
- 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:
- Be able to walk at least 100 meters independently (without assistive devices).
- Have an NSAA total score between 16 and 29, inclusive.
- Participant has received age-appropriate routine childhood immunizations per the local country's national immunization schedule.
- The participant's parent(s)/LAR(s) are willing and able to provide written informed consent prior to the initiation of any trial-specific procedures; where applicable, the participant must provide written or verbal assent in accordance with local regulations.
- The participant and their parent(s)/LAR(s) are willing to participate in a LTFU study after the completion of this trial.
Exclusion Criteria9
- Prior treatment with any gene therapy, gene editing therapy, or cell-based therapy at any time.
- Receipt of any investigational medication or experimental therapy within 6 months prior to Day 1.
- Prior or ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose or inability or unwillingness to refrain from initiating or resuming these therapies for at least 5 years following gene therapy administration.
- Prior ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose.
- Concurrent enrollment in another clinical trial, unless it is observational (non-interventional).
- A positive test for antibodies to AAV9
- A participant has any condition that would contraindicate treatment with immunosuppression.
- Participants with pathogenic mutations in exons 1-44 and/or exons 56-79.
- Evidence of cardiomyopathy or clinically significant left ventricular dysfunction, defined as LVEF <50% on screening echocardiogram.
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Interventions
Participants will receive a single dose of PBGENE-DMD
Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07429240