NCT02286089

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

Phase I/IIa Dose Escalation Safety and Efficacy Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelium Cells Transplanted Subretinally in Patients With Advanced Dry-Form Age-Related Macular Degeneration (Geographic Atrophy)

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 50 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Age-Related Macular Degeneration

Interventions

Type	Name	Description
BIOLOGICAL	OpRegen	Targeted dose of 50,000 - 200,000 cells will be delivered into the subretinal space.

Timeline

• Start date: 2015-04-01
• Primary completion: 2021-12-31
• Completion: 2031-01-31
• First posted: 2014-11-07
• Last updated: 2026-01-29
• Results posted: 2023-03-06

Locations

7 sites across 2 countries: United States, Israel

Regulatory

• FDA-regulated drug study