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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04123626 | A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene | PHASE1 | ACTIVE NOT_RECRUITING | 11 | — | — | Oct 7, 2019 | Jun 7, 2022 | May 6, 2022 | 5 | United States |
Incidence and severity of ocular adverse events scored based on CTCAC in the study and fellow eye
Incidence and severity of non-ocular adverse events scored based on CTCAC in the study and fellow eye
| Arm | Type | Description |
|---|---|---|
| QR-1123 Single dose - dose level 1 | EXPERIMENTAL | Open label Single dose cohort: dose level 1 |
| QR-1123 Single dose - dose level 2 | EXPERIMENTAL | Open label Single dose cohort: dose level |
| QR-1123 Single dose - dose level 3 | EXPERIMENTAL | Open label Single dose cohort: dose level 3 |
| QR-1123 Single dose - dose level 4 | EXPERIMENTAL | Open label Single dose cohort: dose level 4 |
| QR-1123 Single dose - dose level 5 | EXPERIMENTAL | Open label Single dose cohort: dose level 5 |
| Repeat dose cohort 1 | EXPERIMENTAL | Double-masked, randomized, sham controlled, Repeat dose cohort. Dose levels will be determined following DMC review of obtained safety and efficacy data. |
| Name | Type | Description |
|---|---|---|
| QR-1123 | DRUG | unilateral IVT injection |
| Sham procedure | OTHER | Sham procedures (i.e. no penetration of the globe) closely mimic the active injection and serve to mask subjects to treatment assignment |
Main Inclusion Criteria: 1. Male or female, ≥ 18 years of age. 2. Clinical presentation consistent with adRP, based on ophthalmic examinations. 3. Impairment on VF in the opinion of the Investigator, as determined by perimetry. 4. A molecular diagnosis of autosomal dominant form of RP with the P23H...