Phase 1/2a Study of PBGENE-DMD for Duchenne Muscular Dystrophy
This study is testing a new treatment called PBGENE-DMD for Duchenne Muscular Dystrophy (DMD). PBGENE-DMD is given as a single intravenous (IV) dose. This treatment is for boys aged 2 to 7 years old who have a specific type of DMD mutation (changes in their DNA) that can be helped by PBGENE-DMD. The main goal of this Phase 1/2a study is to see how safe PBGENE-DMD is and if it causes any side effects. Researchers will also look for early signs of how well it works. Up to 18 participants will be enrolled in this study. The study is currently unclear regarding its recruitment status.
- Study design
- This is a Phase 1/2a, open-label study, meaning both you and the study team will know what treatment you are receiving. It will enroll up to 18 male participants.
- What's involved
- You would receive a single IV dose of PBGENE-DMD. You would also receive medications around the time of dosing to help manage potential immune responses. Your total participation in the trial would be approximately 130 weeks.
- Compensation
- Not stated in the trial record.
- Follow-up
- Your safety will be monitored for 104 weeks (about two years) after receiving the PBGENE-DMD dose.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
At a glance
Conditions
Where it's being run
2 sites across 2 statesWho to contact
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What this trial measures
- Incidence, severity, and causality of treatment-emergent adverse events and serious adverse eventsFrom Dosing through Week 104
Adverse events and serious adverse events that occur or worsen after initiation of the investigational treatment