NCT06394232

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

A Phase 1/2a Multi-Center, Dose-Escalation Study to Evaluate the Safety & Efficacy of Eyecyte-RPE™ When Administered as a Single-dose Subretinal Injection in Subjects With Geographic Atrophy Secondary to Dry Age-related Macular Degeneration

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 54 (estimated)
• Sponsor: Eyestem Research Pvt. Ltd. · Industry
• Sex: All
• Age: 50 Years – 100 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Retinal Disease
• Macular Degeneration
• Age-Related Macular Degeneration
• Geographic Atrophy
• Eye Diseases

Interventions

Type	Name	Description
DRUG	Eyecyte-RPE™	Eyecyte-RPE™ is a suspension of hiPSCs (human induced Pluripotent Stem Cells) derived Retinal Pigment Epithelial Cells

Timeline

• Start date: 2024-06-04
• Primary completion: 2025-04-01
• Completion: 2030-12-01
• First posted: 2024-05-01
• Last updated: 2024-09-24

Locations

3 sites across 1 country: India