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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05926583 | A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa | PHASE3 | ACTIVE NOT_RECRUITING | 4 | — | — | Sep 12, 2023 | Nov 12, 2030 | Jun 5, 2026 | 1 | Japan |
An AE is any untoward medical occurrence in a clinical study participant administered a investigational or non-investigational medicinal product. An AE does not necessarily have a causal relationship with the treatment.
Number of participants with abnormalities in clinical laboratory assessment (hematology and serum chemistry) will be reported.
| Arm | Type | Description |
|---|---|---|
| Group 1: AAV5-hRKp.RPGR Low Dose | EXPERIMENTAL | Participants will receive bilateral subretinal administration of AAV5-hRKp.RPGR low dose, with surgical delivery to the 2 eyes performed within 7 to 21 days apart. |
| Group 2: AAV5-hRKp.RPGR High Dose | EXPERIMENTAL | Participants will receive bilateral subretinal administration of AAV5 hRKp.RPGR high dose, with surgical delivery to the 2 eyes performed within 7 to 21 days apart. |
| Name | Type | Description |
|---|---|---|
| AAV5-hRKp.RPGR | GENETIC | AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure. |
Inclusion Criteria: * Participants who are Japanese male or female aged 5 years or older * Participants diagnosed as X-linked retinitis pigmentosa (XLRP) (generalized rod-cone dystrophy) associated with pathogenic or likely pathogenic variants in the retinitis pigmentosa guanosine triphosphatase re...