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 range
2 Years – 7 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence, severity, and causality of treatment-emergent adverse events and serious adverse events
. 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.
. 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.