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) |
2024-04-06 |
| End date (estimated) |
2030-12-31 |
| 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]. |
|
Administrative Information |
| NCT number |
NCT06394232 |
| ICTRP weblink |
https://trialsearch.who.int/Trial2.aspx?TrialID=NCT06394232 |
| Other study identifiers |
| Name |
CTRI/2024/04/065639 |
| Description |
The Clinical Trials Registry- India (CTRI) |
|
| Source weblink |
https://clinicaltrials.gov/study/NCT06394232 |
| Public contact |
| Email |
jogin.desai@eyestem.com |
| Public email |
jogin.desai@eyestem.com |
| First name |
Jogin |
| Last name |
Desai |
| Phone |
+91 7676235515 |
| Country |
|
|
| Sponsors |
Eyestem Research Pvt. Ltd.
|
Cells |
| 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.; This extended description was generated by ChatGPT and reviewed by the CellGuide team, who added references, and by the CL editors, who approved it for inclusion in CL. It may contain information that applies to only to some subtypes and species, and so should not be considered definitional.
Retinal pigment epithelial (RPE) cells form a single layer of cells at the back of the eye sandwiched between the neurosensory retina and the choroid, playing a significant role in maintaining vision health. These pigment-laden cells are highly specialized and perform an array of metabolic and transport functions essential for the maintenance of the photoreceptor cells (rods and cones) in the retina. The pigmentation of RPE cells actively aids in the absorption of excess light and the prevention of light scattering, thus enhancing the eye's optical properties.
The retinal pigment epithelium forms a key part of the blood/retina barrier. The cells have long sheet-like microvilli on their apical membrane that project into the light-sensitive outer segments of the photoreceptors, forming a close structural interaction. The basolateral membrane of the RPE interacts with the underlying Bruch’s membrane, which separates the RPE cells from fenestrated endothelium of the choriocapillaris.
RPE cells support the photoreceptor by providing them with oxygen and nutrients (such as glucose, retinol and fatty acids) and removing waste products. They also recycle the visual pigment, in a process called the "visual cycle", where the RPE cells play a vital role in the regeneration of visual pigment (11-cis retinol) following the absorption of light. This is essential for the maintenance of photoreceptor excitability.
Beyond this, RPE cells take part in the phagocytosis process, where they digest the shed ends of photoreceptor outer segments, thus, preventing the build-up of waste residue that could otherwise harm retinal health. They also secrete various factors, including growth factors required to maintain the structural integrity of choriocapillaris endothelium and photoreceptors, as well as immunosuppressive factors that play an important role in establishing the immune privilege of the eye. |
|
Recruitment |
| Recruitment Status |
Recruiting |
| Estimated number of participants |
54 |
