Table 3 Stargardt’s macular dystrophy clinical trials
Trial | Disease | Sample size | VA inclusion criteria | Cell derivation and preparation | Transplantation approach | Immuno-suppression | VA outcomes | Adverse events (AE) |
|---|---|---|---|---|---|---|---|---|
South Korean Phase 1/2a NCT01674829 | SMD/ NNV-AMD | 2 SMD | ≤ 20/320 | hESC-RPE suspension | PPV and subretinal delivery | Tacrolimus and MMF | Improved VA in 2/2 | Herpetic vesicles on skin in 1/2 |
MA09-hRPE cell line Phase 1/2a NCT01469832 | SMD | 12 (3 dose escalation cohorts) | ≤ 20/400 | hESC-RPE suspension | PPV and subretinal delivery | Tacrolimus and MMF | Stable vision in 12/12 | Retinal tears and dialysis in 1 /15 Subretinal hemorrhage in 2/15 Vitreous hemorrhage in 1/15 Immunosuppression side effects (HSV, lethargy, headache, nausea) in 5/15 |
Advanced Cell Technology Phase 1/2a NCT01345006 | SMD/ NNV-AMD | 9 SMD (3 dose escalation cohorts) | ≤ 20/200 | hESC-RPE suspension | PPV and subretinal delivery | Tacrolimus and MMF | > 15 letter improvement in 3/9 Stable vision in 3/9 > 10 letter decrease in 1/9 | Preretinal pigmentation in 1/9 Endophthalmitis 1/9 Vitreous inflammation with vitreous band formation in 1/9 |