PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCT… (NCT07429240) | Clinical Trial Compass
RecruitingPhase 1/2
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
United States18 participantsStarted 2026-04-24
Plain-language 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
Age range2 Years – 7 Years
SexMALE
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Inclusion criteria
✓. 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.
✓. 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).
✓. Be able to walk at least 100 meters independently (without assistive devices).
✓. Have an NSAA total score between 16 and 29, inclusive.
Exclusion criteria
✕. 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.
What they're measuring
1
Incidence, severity, and causality of treatment-emergent adverse events and serious adverse events
. 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.