Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07429240 | PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD) | PHASE1 | RECRUITING | 18 | — | — | Apr 24, 2026 | Dec 1, 2029 | Apr 30, 2026 | 1 | United States |
Adverse events and serious adverse events that occur or worsen after initiation of the investigational treatment
| Arm | Type | Description |
|---|---|---|
| Experimental- Part 1 (Initial Safety) & Part 2 (Expansion) cohort | EXPERIMENTAL | The trial is planned to enroll participants into 2 parts as follows: * Part 1 (Initial Safety) A total of up to 6 participants may be enrolled. * Part 2 (Expansion) Up to 12 participants |
| Name | Type | Description |
|---|---|---|
| PBGENE-DMD (IV) | BIOLOGICAL | Participants will receive a single dose of PBGENE-DMD |
Inclusion Criteria: 1. Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent 2. Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 \[inclusive\]) 3. Clinical phenotype consistent with DMD in the opinion of the Investigator 4. Ability to co...