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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04517149 | 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) | PHASE1 | ACTIVE NOT_RECRUITING | 21 | — | — | Jun 9, 2020 | May 1, 2029 | Mar 21, 2025 | 8 | United States |
| Arm | Type | Description |
|---|---|---|
| 4D-125 Dose Exploration | EXPERIMENTAL | Dose 1 and Dose 2 4D-125 will be administered at the assigned dose level as a single dose, IVT injection on Day 1. The contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent. |
| 4D-125 Dose Expansion | EXPERIMENTAL | 4D-125 will be administered at the assigned dose level as a single dose, IVT injection on Day 1 in both adults and pediatric participants. For adult participants only, the contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent. |
| Observational | OTHER | Natural History |
| Name | Type | Description |
|---|---|---|
| 4D-125 IVT Injection | BIOLOGICAL | 4D-125 drug product developed for gene therapy, which comprises an AAV capsid variant (4D-R100) carrying a codon-optimized human Retinitis Pigmentosa GTPase Regulator transgene. |
| Observational | OTHER | Natural History |
Natural History Key Inclusion Criteria: * Male, ≥ 6 years of age at the time of informed consent * Hemizygous non-syndromic RPGR mutation confirmed by genetic testing Interventional Key Inclusion Criteria: * Male, ≥12 years of age * Hemizygous non-syndromic RPGR mutation confirmed by genetic test...