Safety and Efficacy Study of OpRegen for Treatment of Advanced Dry-Form Age-Related Macular Degeneration

General Information
Summary	The main objective of the study is evaluation of the safety and tolerability of OpRegen - human embryonic stem cell-derived retinal pigment epithelial (RPE)cells. The study will also include initial exploration of the ability of transplanted OpRegen cells to engraft, survive, and moderate disease progression.
Description	OpRegen® is a cell-based product composed of retinal pigment epithelial (RPE) cells, derived from human embryonic stem cells (hESC) and administered as a cell suspension either in ophthalmic Balanced Salt Solution Plus (BSS Plus) or in CryoStor® 5 (Thaw-and-Inject, TAI). This is a Phase I/IIa, dose-escalation, evaluating safety and tolerability of OpRegen transplantation to patients with progressive dry-AMD. The study includes also initial exploration of efficacy. A total of approximately 24 subjects will be enrolled. The subjects should be 50 years of age and older, with non-neovascular (dry) AMD, who have funduscopic findings of GA in the macula, with absence of additional concomitant ocular disorders. The subjects will be divided into four cohorts, according to their best corrected visual acuity (BCVA) and administered OpRegen dose.
Clinical trials phase	Phases 1/2
Start date (estimated)	2015-04-01
End date (estimated)	2025-07-01
Clinical feature	Label age related macular degeneration Link http://purl.obolibrary.org/obo/DOID_10871 Description A degeneration of macula and posterior pole that is characterized by a loss of vision in the center of the visual field (the macula) resulting from damage to the retina and resulting in blurring of the sharp central vision.; OMIM mapping confirmed by DO. [SN].
Publications	Derivation of xeno-free and GMP-grade human embryonic stem cells--platforms for future clinical applications Directed differentiation of human embryonic stem cells into functional retinal pigment epithelium cells Long-Term Efficacy of GMP Grade Xeno-Free hESC-Derived RPE Cells Following Transplantation
Administrative Information
NCT number	NCT02286089
ICTRP weblink	https://trialsearch.who.int/Trial2.aspx?TrialID=NCT02286089
Other study identifiers	Name GR44172
Source weblink	http://clinicaltrials.gov/ct2/show/NCT02286089
Public contact	Email patientcare@roche.com Public email patientcare@roche.com
Sponsors	Hoffmann-La Roche
Cells
Source pluripotent stem cell lines	HADe008-A
Which differentiated cell type is used	Label retinal pigment epithelial cell Link http://purl.obolibrary.org/obo/CL_0002586 Description An epithelial cell of the retinal pigmented epithelium.
Recruitment
Recruitment Status	Active, not recruiting
Estimated number of participants	24