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Retinal DiseasePhase 1/2NCT06394232

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

A Phase 1/2 study testing Eyecyte-RPE™ in people with Retinal Disease and Macular Degeneration, run by Eyestem Research Pvt. Ltd. at 3 sites in India.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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). Read more about this study

Who is it for?
Ages 50 to 100 years
What is being tested?
Eyecyte-RPE™
Could I receive a placebo?
No, everyone receives an active treatment
How many people
54 participants
Where
3 sitesIndia
Study dates
4 Jun 2024to Dec 2030
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 to 100 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Men and women ≥ 50 years of age at Screening.
  • Diagnosis of Geographic Atrophy secondary to d-AMD
  • Have Best Corrected Visual Acuity (BCVA) equal to or less than 20/200 Snellen (ETDRS letter score ≤ 35) in the study eye at screening.
  • Phase 1 ≤ 20/200 and
  • Phase 2a ≥ 20/64 (ETDRS letter score 60) in the study eye at Screening.
  • Vision in the unoperated eye must be better or equal to vision in the study eye.
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  • Willing, committed, and able to return for ALL clinic visits and complete all study related procedures.
  • Be medically suitable to undergo anesthesia, vitrectomy and subretinal injection in the opinion of the Investigator.
  • Be medically suitable for immunosuppression therapy in accordance with the requirements of this protocol in the opinion of the Investigator.
  • 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)
  • Willing to provide signed Informed Consent prior to any procedures being performed at Visit 1, Screening.
  • Negative for HIV, HbsAg, HCV, TB
  • The GA lesion must meet the following criteria as determined by the central reading center's assessment of Fundus Autofluorescence (FAF) imaging at screening:
  • Total GA area must be ≥ 1.25 and ≤ 17.5 mm2 (0.5 and 7 disk areas [DA] respectively)
  • 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.
  • At least one of the lesions has to be sub-foveal.

Who cannot join

  • Have evidence of neovascular AMD in either eye by clinical examination, fluorescein angiography or optical coherence tomography.
  • 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.
  • 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 study eye.
  • Axial myopia greater than -6 diopters or axial length more than 26 mm.
  • Have a decrease in BCVA in the study 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.).
  • Have the presence of retinal pigment epithelial tears or rips involving the macula in the study eye at screening.
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  • Have a history or evidence of vitreous hemorrhage in the study eye.
  • Have a history or clinical evidence of severe diabetic retinopathy, diabetic macular edema, retinal vein occlusion or any other vascular disease affecting the retina in the study eye.
  • Have had a prior pars plana vitrectomy in the study eye.
  • Have a history of retinal detachment or treatment or surgery for retinal detachment in the study eye.
  • Have history of a macular hole in the study eye.
  • Have had any other ocular surgery (except cataract) within 2 months or Yttrium Aluminum Garnet (YAG) laser capsulotomy in the study eye in the past 4 weeks.
  • Have had a prior trabeculectomy or other filtration surgery in the study eye.
  • History of any form of glaucoma in the study eye.
  • Patients with ocular pathology, particularly that of retina (other than AMD).
  • Have active intraocular inflammation or a history or evidence of uveitis in either eye.
  • 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, Screening in either eye.
  • Have a history of scleromalacia in either eye.
  • Have had previous therapeutic radiation in the study eye.
  • Have a history of corneal transplant or corneal dystrophy.
  • Have any concurrent ocular condition in the study 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.
  • Have a history of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding 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.
  • Have participated as a subject in any clinical study within 6 months prior to Day 0, Baseline.
  • Have a known serious allergy to fluorescein sodium for injection in angiography, Povidone Iodine or any of the other medications required for anesthesia or the subretinal injection procedure.
  • Be a female who is pregnant, breastfeeding, or of childbearing potential, unwilling to practice adequate contraception throughout the study at Screening and Baseline.
  • Currently receiving aspirin, aspirin containing products and/or any other coagulation modifying drugs which cannot be discontinued 7 days prior to surgery.
  • Have any systemic condition that would qualify the subject as being immunocompromised (e.g., severely uncontrolled diabetes, cancer).
  • Patients with Optic Atrophy

Trial locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Dr Jogin Desai, MBBS contact

+91 7676235515

jogin.desai@eyestem.com

Dr Rajani Battu, MBBS,MS,DNB,FRCS(Edin),PhD contact

+91 7676235515

rajani.battu@eyestem.com

India3 sites, 3 recruiting
  • All India Institute of Medical Sciences

    Delhi, New Delhi, 110029

    Dr.Rajpal, MBBS,MD

    Recruiting
  • L V Prasad Eye Institute

    Hyderabad, Telangana, 500034

    Dr.Raja Narayanan, MBBS,MS

    Recruiting
  • Shri Ganapati Netralaya

    Jālna, Maharashtra, 431203

    Dr.Rushikesh Arunrao, MBBS,DNB

    Recruiting

Other trials in India

About this study

Official title: 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

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.

Read the 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.

Being tested
Eyecyte-RPE™
Keywords
Age Related Macular Degeneration, Geographic Atrophy, Visual Impairment, Subretinal injection, Stem cells, Retinal Pigment Epithelial Cells
Medical subject headings
Retinal Diseases, Macular Degeneration, Geographic Atrophy, Eye Diseases, Vision Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Eyecyte-RPE™Drug

    Also called Subretinal Injection, Oral Tacrolimus, Oral Mycophenolate Sodium

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

Study groups in NCT06394232
Study groupRoleWhat they receive
Dose Level 1-Cohort 1ExperimentalSubjects who are allocated to Dose Level 1-Cohort 1 will receive 100,000 cells of Eyecyte-RPE™
Receives: Drug: Eyecyte-RPE™
Dose Level 2-Cohort 2ExperimentalSubjects who are allocated to Dose Level 2-Cohort 2 will receive 200,000 cells of Eyecyte-RPE™
Receives: Drug: Eyecyte-RPE™
Dose Level 3-Cohort 3ExperimentalSubjects who are allocated to Dose Level 3-Cohort 3 will receive 300,000 cells of Eyecyte-RPE™
Receives: Drug: Eyecyte-RPE™

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Fundus Autofluorescence-

    Overall change in the geographic atrophy (GA) lesion area of the study eye over time using Fundus Autofluorescence (FAF)

    Measured: Upto 12 months

Secondary outcomes (5)
  1. Best Corrected Visual Acuity (BCVA)-Mean change from baseline in BCVA score in study eye and fellow eye.

    Change from baseline over time in BCVA as measured by Early Treatment Diabetic Retinopathy Study (ETDRS) chart. The BCVA score (in letter units) will be reported.

    Measured: Upto 12 months

  2. Spectral-Domain Optical Coherence Tomography (SD-OCT)-Mean change from baseline in the Geographic Atrophy (GA) area in study eye and fellow eye

    Overall change in the GA lesion area of the study eye over time using SD-OCT

    Measured: Upto 12 months

  3. Fundus Photography- Changes between study eye and fellow eye

    Correlation assessment between quantitative metrics derived from Color fundus photography images

    Measured: Upto 12 months

  4. Microperimetry-Mean change from baseline in mean sensitivity

    Change from initial assessment over time in retinal sensitivity, as assessed by Microperimetry (MP)

    Measured: Upto 12 months

  5. National Eye Institute-Visual Function Questionnaire Quality of Life score

    Change over time in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life (QoL) score

    Measured: Upto 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 May 2024
  2. Study start4 Jun 2024
  3. Primary completionApr 2025 (estimated)
  4. Study completionDec 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Model description
Subjects will be enrolled into 3 sequential, ascending dose-level (DL) cohorts to enroll a maximum of 18 subjects. There will be three Cohorts 1. DL1-Cohort 1: Eyecyte-RPE™ 100,000 cells 2. DL2-Cohort 2: Eyecyte-RPE™ 200,000 cells 3. DL3-Cohort 3: Eyecyte-RPE™ 300,000 cells
Masking
None (open label)
Enrolment
54 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06394232
Sponsor's protocol ID
ERPL-CTP-001
Status last verified
Sep 2024
First submitted
15 Apr 2024
Last updated
24 Sep 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Soundararajan L, Surendran H, Patlolla N, Battu R, Stoddard J, Arrizabalaga S, Liu Z, Lingam G, Su X, Ryals RC, Pal R. Allogeneic RPE cell suspension manufactured at scale demonstrating preclinical safety and efficacy led to IND approval. NPJ Regen Med. 2025 Apr 19;10(1):19. doi: 10.1038/s41536-025-00407-0.

    PubMed 40253438

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Eyecyte-RPE™ in people with Retinal Disease and Macular Degeneration, run by Eyestem Research Pvt. Ltd. at 3 sites in India.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 16 inclusion and 28 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 24 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in India. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Dr Jogin Desai, MBBS (+91 7676235515, jogin.desai@eyestem.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Fundus Autofluorescence- (time frame: Upto 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06394232, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2024.

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