Table 2 Neovascular age-related macular degeneration trials
Trial | Disease | Sample size | VA inclusion criteria | Cell derivation and preparation | Transplantation approach | Immuno-suppression | VA outcomes | Adverse events (AE) |
|---|---|---|---|---|---|---|---|---|
RPE Sheet Case Report UMIN000011929 | NV-AMD | 2 | ≤ 20/200 | iPSC-RPE sheet | PPV and subretinal delivery | None | Stable vision in 1/2 | Cystoid macular edema in 1/2 Transplantation aborted because of concerns about genetic changes that occurred in the iPSCs and iPSC-derived RPE cells in 1/2 |
HLA-matched iPSC-RPE UMIN000026003 | NV-AMD | 5 | ≤ 20/200 | iPSC-RPE suspension from HLA homozygous donor | PPV and subretinal delivery | None | > 10 letter gain in 2/5 Stable vision in 3/5 | No severe AEs Mild inflammation in 2/5 Epiretinal membrane in 1/5 Aseptic endophthalmitis in 1/5 |
Q-CTS-hESC-2 NCT02749734 | NV-AMD | 3 | ≤ 20/400 | hESC-RPE suspension | PPV and subretinal delivery | Tacrolimus, MMF, Prednisone | Improved VA in 2/3 Stable vision in 1/3 | No severe AEs |
SHEF-1.3 hESC patch NCT01691261 | NV-AMD | 2 | ≤ 20/120 | hESC-RPE monolayer | PPV and subretinal delivery | Prednisone, Fluocinolone implant | > 20 letter gain in 2/2 | Retinal detachment with proliferative vitreoretinopathy in 1/2 Exposure of fluocinolone implant suture in 1/2 |