CTRI/2024/04/065639 [Registered on: 15/04/2024] Trial Registered Prospectively
Last Modified On:
24/03/2026
Post Graduate Thesis
No
Type of Trial
Interventional
Type of Study
Drug Biological Stem Cell Therapy
Study Design
Randomized, Parallel Group, Multiple Arm Trial
Public Title of Study
Study to Understand Safety Efficacy of Novel Stem Cell Formulation to Treat Late Stage Dry Age-related Macular Degeneration (d-AMD)
Scientific Title of Study
A Phase 1/2a Multi-Center Dose-Escalation Dose-Expansion Study to
Evaluate the Safety and Efficacy of Eyecyte-RPEâ„¢ when administered as a
Single-dose Subretinal Injection in Subjects with Geographic Atrophy
GA Secondary to Dry Age-related Macular Degeneration d-AMD
Details of Principal Investigator or overall Trial Coordinator (multi-center study) Modification(s)
Name
Dr Rajpal
Designation
Professor of Ophthalmology
Affiliation
Dr. Rajendra Prasad Centre for Ophthalmic Sciences, All India Institute of Medical Sciences
Address
Ansari Nagar, New Delhi 110029, India.
New Delhi DELHI 110029 India
Phone
9818598899
Fax
Email
vohrarajpal@gmail.com
Details of Contact Person Scientific Query
Name
Dr Rajani Battu
Designation
Co-founder and Chief Medical Officer
Affiliation
Eyestem Research Pvt. Ltd.
Address
Centre for Cellular and Molecular Platforms (C-CAMP)
NCBS-TIFR, GKVK Post, Bellary Road,
Bengaluru, Karnataka 560065 (INDIA)
Bangalore KARNATAKA 560065 India
Phone
9900265081
Fax
Email
rajani.battu@eyestem.com
Details of Contact Person Public Query
Name
Dr Jogin Desai
Designation
Founder and Chief Executive Officer
Affiliation
Eyestem Research Pvt. Ltd.
Address
Centre for Cellular and Molecular Platforms (C-CAMP)
NCBS-TIFR, GKVK Post, Bellary Road,
Bengaluru, Karnataka 560065 (INDIA)
Bangalore KARNATAKA 560065 India
Phone
9739383877
Fax
Email
jogin.desai@eyestem.com
Source of Monetary or Material Support
Eyestem Research Pvt. Ltd.
Centre for Cellular and Molecular Platforms (C-CAMP)
NCBS-TIFR, GKVK Post, Bellary Road,
Bengaluru, Karnataka 560065 (INDIA)
Primary Sponsor
Name
Eyestem Research Pvt. Ltd.
Address
Centre for Cellular and Molecular Platforms (C-CAMP)
NCBS-TIFR, GKVK Post, Bellary Road,
Bengaluru, Karnataka 560065 (INDIA)
Aravind Eye Hospital and Postgraduate Institute of Ophthalmology
No 1, Anna Nagar, Madurai -625020, Tamil Nadu, India Madurai TAMIL NADU
9443490920
cauveryeye@gmail.com
Dr Rajpal
Dr. Rajendra Prasad Centre for Ophthalmic Sciences, All India Institute of Medical Sciences
Ansari
Nagar, New Delhi 110029 India
New Delhi DELHI
9818598899
vohrarajpal@gmail.com
Dr Vivek Pravin Dave
LV Prasad Eye Institute
Hyderabad Eye Research Foundation (HERF),
LV Prasad Eye Institute, Kallam Anji Reddy Campus, Banjara Hills, Hyderabad- 500034,
Telangana, India Hyderabad TELANGANA
7680859900
vivekdave@lvpei.org
Dr Muna Bhende
Sankara Nethralaya
Medical Research Foundation, 41/18, College Road, Nungambakkam, Chennai-
600006, Tamil Nadu, India Chennai TAMIL NADU
(1) ICD-10 Condition: H353||Degeneration of macula and posterior pole,
Intervention / Comparator Agent
Type
Name
Details
Intervention
Eyecyte-RPEâ„¢
Eyecyte-RPEâ„¢ is a suspension of hiPSCs (human induced Pluripotent Stem Cells)
derived Retinal Pigment Epithelial Cells.
Eyecyte-RPEâ„¢, developed by Eyestem can help to replace the damaged or lost RPE cells and potentially enable tissue regeneration in the diseased retina
1. Men and women greater than or equal to 50 years of age at Screening.
2. Diagnosis of Geographic Atrophy secondary to d-AMD.
3. Have Best Corrected Visual Acuity (BCVA) in the study eye as follows at screening.
a. Phase 1 less than or equal to 20/200 and
b. Phase 2a greater than or equal to 20/800 and less than or equal to 20/100 in the study eye at Screening.
4. Vision in the unoperated eye must be better or equal to vision in the study eye.
5. Willing, committed, and able to return for ALL clinic visits and complete all study related procedures.
6. Be medically suitable to undergo anesthesia, vitrectomy and subretinal injection in the opinion of the Investigator.
7. Be medically suitable for immunosuppression therapy in accordance with the requirements of this protocol in the opinion of the Investigator.
8. Able to read (or if unable to read due to visual impairment, be read to verbatim by the person administering the informed consent or a family member) and understand, and willing to sign the informed consent form (ICF).
9. Willing to provide signed Informed Consent prior to any procedures being performed at Visit 1.
10. Participants should have no history and current reactive status for HIV, HBsAg, or HCV infection, as confirmed by laboratory tests. No active signs of TB as confirmed by Chest X-ray.
11. The GA lesion must meet the following criteria as determined by the assessment of Fundus Autofluorescence (FAF) imaging at screening:
a. Total GA area must be greater than or equal to 1.25 and less than or equal to 17.5 mm2 (0.5- and 7-disc areas [DA] respectively).
b. The entire GA lesion must be completely visualized on the macula centered image and must be able to be imaged in its entirety and not contiguous with any areas of peripapillary atrophy.
c. At least one of the lesions must be partially sub-foveal.
ExclusionCriteria
Details
1. Have evidence of neovascular AMD in either eye by clinical examination, fluorescein angiography, or optical coherence tomography.
2. Have GA secondary to a condition other than AMD such as Stargardt disease, cone rod dystrophy, or toxic maculopathies like Chloroquine maculopathy in either eye.
3. Have any evidence of active or inactive choroidal neovascularization (CNV) due to other causes such as ocular histoplasmosis syndrome, angioid streaks, choroidal rupture, uveitis, punctate inner choroidopathy, or multifocal choroiditis in the either eye. (only Study eye will be considered for Phase 1)
4. Axial myopia is greater than -6 diopters or axial length more than 26 mm
5. Have a decrease in BCVA in the either eye due to causes other than GA (e.g., pigment abnormalities, dense sub foveal hard exudates, previous vitreoretinal surgery, retinal dystrophies, non-retinal conditions, visually significant cataract, macular ischemia, etc.). (only Study eye will be considered for Phase 1)
6. Have the presence of retinal pigment epithelial tears or rips involving the macula in the either eye at screening. (only Study eye will be considered for Phase 1)
7. Have a history or evidence of vitreous hemorrhage in the either eye. (only Study eye will be considered for Phase 1)
8. Have history or clinical evidence of severe diabetic retinopathy, diabetic macular edema, retinal vein occlusion, or any other vascular disease affecting the retina in the either eye. (only Study eye will be considered for Phase 1)
9. Have had a prior pars plana vitrectomy in the either eye. (only Study eye will be considered for Phase 1)
10. Have a history of retinal detachment or surgery for retinal detachment in the either eye. (only Study eye will be considered for Phase 1)
11. Have history of a macular hole in either eye. (only Study eye will be considered for Phase 1)
12. Have had any other ocular surgery within 2 months or Yttrium Aluminum Garnet (YAG) laser capsulotomy in the either eye in the past 4 weeks. (only Study eye will be considered for Phase 1)
13. Have had a prior trabeculectomy or other filtration surgery in the either eye. (only Study eye will be considered for Phase 1)
14. History of uncontrolled glaucoma in the either eye. (History of any form of glaucoma in study eye will be considered for Phase 1)
15. Patients with ocular pathology, particularly that of retina (other than AMD).
16. Have active intraocular inflammation or a history or evidence of uveitis in the either eye.
17. Have active ocular or periocular infection in either eye, or a history of any ocular or periocular infection within the 2 weeks prior to Visit 1 in either eye.
18. Have a history of scleromalacia in either eye.
19. Have had previous therapeutic radiation in the either eye. (only Study eye will be considered for Phase 1)
20. Have a history of corneal transplants or corneal dystrophy.
21. Have any concurrent ocular conditions in the either eye which, in the opinion of the investigator, could either increase the risk to the subject beyond what is to be expected from standard procedures of intraocular injection, or which otherwise may interfere with the injection procedure or with evaluation of efficacy or safety. (only Study eye will be considered for Phase 1)
22. Have a history of other diseases, physical examination findings, or clinical laboratory findings giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that might affect interpretation of the results of the study or render the subject at high risk for treatment complications.
23. Have participated as a subject in any clinical study within 6 months prior to the Screening visit.
24. Have a known serious allergy to fluorescein sodium for injections in angiography, Povidone Iodine or any of the other medications required for anesthesia or the subretinal injection procedure.
25. Be a female who is pregnant, breastfeeding, or of childbearing potential, unwilling to practice adequate contraception throughout the study at Screening and Baseline.
26. Currently receiving aspirin, aspirin containing products and/or any other coagulation modifying drugs which cannot be discontinued 7 days prior to surgery (Not Applicable for Phase 2a).
27. Have any systemic condition that would qualify the subject as being immuno compromised (e.g., severely uncontrolled diabetes, cancer).
28. Patients with Optic Atrophy.
29. Patients with only one functional eye (one-eyed patients) (Applicable for Phase 2a)
30. Subjects with a history of cataract surgery in the study eye within the last 3 months, any ocular abnormality in the fellow eye other than dry AMD, or current use of medications known to cause maculopathy. (Applicable for Phase 2a)
Primary Efficacy Endpoint - Phase 1
a Fundus Autofluorescence
Phase 2
a BCVA
Phase 1
At Screening (≥ 28 days),
During Active Follow-up on the following visits
Days- 1, 7, 14, 28, 42, 56, 70, 84, 120, 150 and 180
During Safety Follow-up on the following visits
Months- 12, 18, 24, 36, 48 and 60
Phase 2
At Screening (≥ 28 days),
During Active Follow-up on the following visits
Days- 1, 7, 28, 56,84, 120, 150 and 180
During Safety Follow-up on the following visits
Months- 9,12, 15,18,21, 24, 36, 48 and 60
Total Sample Size="42" Sample Size from India="42" Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials" Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials"
Individual Participant Data (IPD) Sharing Statement
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
Brief Summary
Age-related Macular Degeneration (AMD) is a leading cause of vision impairment in the elderly and substantially affects the quality of life of an individua. 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 d-AMD. GA is a slow but inexorably progressive disease that causes irreversible blindness over time. In recent years, several new non-invasive tools are being used to enable early diagnosis and follow up in patients with GA, such as Fundus Autofluorescence (FAF), Optical Coherence Tomography (OCT), and OCT Angiography (OCT-A). 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 AMD, which is an unmet medical need globally, Eyestem has been striving to develop a safe and efficacious stem-cell based therapy.
Theoretically, hiPSCs derived Eyecyte-RPEâ„¢, developed by Eyestem can help replace the damaged or lost 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-RPEâ„¢ 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 this that this therapy may have substantial therapeutic value in dry AMD.