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

NCT06394232 | Recruiting Phase 1 DMC

• 1 other identifier: ERPL-CTP-001
• Study Type: interventional
• Target: 54
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

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

Keywords

Age Related Macular Degeneration; Geographic Atrophy; Visual Impairment; Subretinal injection; Stem cells; Retinal Pigment Epithelial Cells

Outcome Measures

Primary Outcomes (1)

• Fundus Autofluorescence-

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

  Upto 12 months

Secondary Outcomes (5)

• Best Corrected Visual Acuity (BCVA)-Mean change from baseline in BCVA score in study eye and fellow eye.

  Upto 12 months

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

  Upto 12 months

• Fundus Photography- Changes between study eye and fellow eye

  Upto 12 months

• Microperimetry-Mean change from baseline in mean sensitivity

  Upto 12 months

• National Eye Institute-Visual Function Questionnaire Quality of Life score

  Upto 12 months

Study Arms (3)

Dose Level 1-Cohort 1

EXPERIMENTAL

Subjects who are allocated to Dose Level 1-Cohort 1 will receive 100,000 cells of Eyecyte-RPE™

Drug: Eyecyte-RPE™

Dose Level 2-Cohort 2

EXPERIMENTAL

Subjects who are allocated to Dose Level 2-Cohort 2 will receive 200,000 cells of Eyecyte-RPE™

Drug: Eyecyte-RPE™

Dose Level 3-Cohort 3

EXPERIMENTAL

Subjects who are allocated to Dose Level 3-Cohort 3 will receive 300,000 cells of Eyecyte-RPE™

Drug: Eyecyte-RPE™

Interventions

Eyecyte-RPE™ DRUG

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

Also known as: Subretinal Injection, Oral Tacrolimus, Oral Mycophenolate Sodium

Dose Level 1-Cohort 1; Dose Level 2-Cohort 2; Dose Level 3-Cohort 3

Eligibility Criteria

• Age: 50 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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).

Sponsors & Collaborators

• Eyestem Research Pvt. Ltd.: lead

Study Sites (3)

Shri Ganapati Netralaya

Jālna, Maharashtra, 431203, India

RECRUITING

All India Institute of Medical Sciences

Delhi, New Delhi, 110029, India

RECRUITING

L V Prasad Eye Institute

Hyderabad, Telangana, 500034, India

RECRUITING

Related Publications (1)

• 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.

  PMID: 40253438 DERIVED

MeSH Terms

Conditions

Retinal Diseases; Macular Degeneration; Geographic Atrophy; Eye Diseases; Vision Disorders

Interventions

Tacrolimus; Mycophenolic Acid

Condition Hierarchy (Ancestors)

Retinal Degeneration; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids

Central Study Contacts

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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

Study Record Dates

• First Submitted: April 15, 2024
• First Posted: May 1, 2024
• Study Start: June 4, 2024
• Primary Completion: April 1, 2025
• Study Completion (Estimated): December 1, 2030
• Last Updated: September 24, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

IN (3)