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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05693142 | AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) | PHASE2 | RECRUITING | 65 | — | — | Jan 4, 2023 | Aug 1, 2028 | Dec 17, 2025 | 17 | United States, Canada |
Evaluate incidences of AEs and SAEs
Proportion of participants whose RGX-202 microdystrophin protein expression determined in their muscle biopsy is ≥ 10%
| Arm | Type | Description |
|---|---|---|
| Part 1: Cohort 1 and 1b: RGX-202 Dose 1 | EXPERIMENTAL | A single IV infusion of RGX-202 at a dose of 1×10\^14 GC/kg body weight |
| Part 1: Cohort 2, 2c;, and Part 2; and Part 3: RGX-202 Dose 2 | EXPERIMENTAL | A single IV infusion of RGX-202 at a dose of 2x10\^14 GC/kg body weight |
| Name | Type | Description |
|---|---|---|
| RGX-202 | GENETIC | RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin |
Part 1 - Key Inclusion Criteria: * The participant's legal guardian(s) is (are) willing and able to provide written, signed informed consent prior to any study-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements. * I...