Safety & Efficacy of Eyecyte-RPE™ in Patients With Geographic Atrophy Secondary to Dry Age-related Macular Degeneration

General Information

Summary The goal of this clinical study is to evaluate the safety and efficacy of novel stem cell formulation in patients having Geographic Atrophy (GA) Secondary to Dry Age-related Macular Degeneration (d-AMD). The main questions it aims to answer are: Safety and tolerability of the novel stem cell formulation Potential efficacy of the novel stem cell formulation Participants will receive a single subretinal injection in their study eye and followed up for safety. This is an India only study and the product is developed indigenously.
Description Age-related Macular Degeneration (AMD) is a leading cause of vision impairment in the elderly and substantially affects the quality of life of an individual. Although the exact pathophysiological mechanisms behind the disease are multifactorial and complex, several genetic and environmental risk factors are associated with AMD, such as age, cigarette smoking, hypertension, abdominal obesity, dietary fat, and low physical activity. Geographic Atrophy (GA) is the late stage of dry AMD(d-AMD). GA is a slow but inexorably progressive disease that causes irreversible blindness over time. Several new non-invasive tools such as Fundus Autofluorescence (FAF), Optical Coherence Tomography (OCT), and OCT Angiography (OCT-A) are being used to enable early diagnosis and follow up in these patients. Despite these advances, there are currently no approved treatments for GA that can replace the damaged RPE, photoreceptors, or outer retinal layers. In recent years, stem cell replacement therapy is being evaluated as an alternative to treat d-AMD. In the pursuit to find a promising solution for d-AMD, which is an unmet medical need globally, Eyestem has been striving to develop a safe and effective stem-cell based therapy. Theoretically, human induced pluripotent stem cells(hiPSCs) derived Eyecyte-RPE™, developed by Eyestem can help replace the damaged or lost retinal pigment epithelial (RPE) cells and potentially enable tissue regeneration in the diseased retina. In addition, stem cells can perform multiple functions, such as immunoregulation, prevention of apoptosis in sensory neurons, and secretion of neurotrophic factors. The latest stem cell transplantation studies performed by other research groups has been able to demonstrate that this therapy has a promising approach to restore visual function in eyes with degenerative retinal diseases. The results of the pre-clinical safety and efficacy studies with Eyecyte-RPE™ have been very encouraging. It has been shown to provide significant beneficial effects on the degenerating retina in animals without any significant safety concerns, suggesting that this therapy may have substantial therapeutic value in d-AMD.
Clinical trials phase Phases 1/2
Start date (estimated) 2024-04-22
End date (estimated) 2030-12-31
Clinical feature
Label age related macular degeneration
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].

Administrative Information

NCT number NCT06394232
ICTRP weblink
Other study identifiers
Name ERPL-CTP-001
Name CTRI/2024/04/065639
Description The Clinical Trials Registry- India (CTRI)
Source weblink
Public contact
Public email
First name Jogin
Last name Desai
Phone +91 7676235515
Sponsors Eyestem Research Pvt. Ltd.


Which differentiated cell type is used
Label retinal pigment epithelial cell
Description An epithelial cell of the retinal pigmented epithelium.


Recruitment Status Not yet recruiting
Estimated number of participants 54