Testing PBGENE-DMD, a gene-editing treatment, in young children with Duchenne muscular dystrophy
Tests the safety of PBGENE-DMD given by infusion in children with Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Key facts
- Where
- Little Rock and St Louis, United StatesSee the 2 sites
- Phase
- Phase 1/2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 7 years old?
Source: NCT07429240, who can take part
Where in United States
- Arkansas Children's HospitalLittle Rock, United StatesTaking people
- Washington University School of MedicineSt Louis, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Precision BioSciences, Inc.
- Trial code
- NCT07429240
- Record updated
- 21 October 2026
- Original description
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.Read full original descriptionShow less
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)
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.
Inclusion: 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: 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; Evidence of cardiomyopathy or clinically significant left ventricular dysfunction, defined as LVEF <50% on screening echocardiogram; Participants with pathogenic mutations in exons 1-44 and/or exons 56-79.