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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04794101 | Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene | PHASE3 | ACTIVE NOT_RECRUITING | 97 | — | — | Dec 4, 2020 | Dec 7, 2029 | Jun 5, 2026 | 28 | United States, Belgium +9 |
Number of participants with ocular and non-ocular adverse events will be assessed.
Number of participants with abnormalities in laboratory assessments will be assessed.
Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.
| Arm | Type | Description |
|---|---|---|
| Deferred Treatment From MGT-RPGR-021 of Intermediate Dose | EXPERIMENTAL | Deferred treatment |
| Already Treated in MGT-RPGR-021 | EXPERIMENTAL | Already treated |
| Deferred Treatment From MGT-RPGR-021 Low Dose | EXPERIMENTAL | Deferred treatment |
| Name | Type | Description |
|---|---|---|
| Genetic: AAV5-hRKp.RPGR Intermediate Dose | BIOLOGICAL | Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group |
| Genetic: AAV5-hRKp.RPGR Low Dose | BIOLOGICAL | Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group |
Inclusion Criteria: * Male or female, 3 years of age or older, has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory. Exclusion Criteria: * None