Table 1 Non-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) |
|---|---|---|---|---|---|---|---|---|
Palucorcel Phase 1/2a NCT01226628 | NNV-AMD | 35 | Phase 1: ≤ 20/200 Phase 2a: ≤ 20/80 | hUTC suspension | Suprachoroidal subretinal delivery | None | > 10 letter gain in 10/29 > 15 letter gain in 7/29 | Retinal detachments in 6/35 Retinal perforations in 13/35 Subretinal delivery aborted due to retinal perforations during the surgical procedure in 2/35 AE related to surgery in 25/33 AE related to surgical delivery system in 19/33 AE related to cell suspension in 5/33 |
Palucorcel Phase 2b NCT02659098 | NNV-AMD | 21 | 20/80 to 20/800 | hUTC suspension | Suprachoroidal subretinal delivery | None | Mean VA change: − 5.7 letters (vs. − 3.7 in control group) > 15 letter gain in 0/21 | No serious AEs, no RDs, no perforations, no significant changes in IOP > 15 letters lost in 3/21 |
CPCB-RPE1 Phase 1/2a NCT02590692 | NNV-AMD | 5 | ≤ 20/200 | hESC-RPE polarized monolayer | PPV and subretinal delivery | Tacrolimus | > 15 letter gain in 1/5 Stable vision in 4/5 | Subretinal hemorrhage requiring bevacizumab treatment in 1/5 Implantation aborted due to subretinal debris 1/5 |
Advanced Cell Technology Phase 1/2a NCT01344993 | NNV-AMD/ SMD | 9 NN-AMD (3 dose escalation cohorts) | ≤ 20/200 | hESC-RPE suspension | PPV and subretinal delivery | Tacrolimus and MMF | > 15 letter gain in 4/9 10–14 letter gain in 2/9 Stable vision in 3/9 | Preretinal pigmentation in 2/9 |
South Korean Phase 1/2a NCT01674829 | NNV-AMD/ SMD | 2 NN-AMD | ≤ 20/320 | hESC-RPE suspension | PPV and subretinal delivery | Tacrolimus and MMF | Improved VA in 1/2 | Discontinuation of immunosuppression due to side effects in 1/2 Choroidal neovascular membrane in 1/2 |