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

h-ESC human embryonic stem cells; HSV herpes simplex virus; MMF mycophenolate mofetil; NNV-AMD non-neovascular age-related macular degeneration; PPV pars plana vitrectomy; RPE retinal pigment epithelium; SMD Stargardt’s macular dystrophy; VA visual acuity