Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration

Recruiting now · Phase 1/Phase 2

Conditions studied: Dry Age-Related Macular Degeneration, Geographic Atrophy

In brief

Background: Age-related macular degeneration is a common eye disease in people over 50. The "dry" form of the disease can worsen into geographic atrophy, causing blind spots. Researchers want to learn if replacing older eye cells with younger ones can help treat this disease. Objective: To test the safety of putting cells inside the eye as a possible future treatment for dry age-related macular degeneration. Eligibility: People ages 55 and older who have geographic atrophy with loss of vision. People who have had "wet" macular degeneration in study eye are NOT eligible. Design: Participants will be screened with: * Medical history * Physical exam * Blood and urine tests * Eye exam * Eye photos * Fluorescein angiography. An intravenous (IV) line is placed in an arm vein. A dye is injected. A camera takes pictures of the dye as it flows through the eyes' blood vessels. * Electroretinography. An electrode is taped to participants' forehead. They sit in the dark. After 30 minutes, numbing eye drops and contact lenses are placed in their eyes. They watch flashing lights. * Tuberculosis test * Chest X-ray * Electrocardiography. Sticky pads are placed on participants' chest to record the heart's electrical activity. Participants will have at least 14 study visits over 5 and a half years. They will repeat screening tests. Participants will have retinal pigment epithelium (RPE) transplantation surgery in one eye. For this, cells from participants' blood are turned into RPE cells. These cells are placed in their eye through a cut in their retina. They will get dilating eye drops, an IV line, and anesthesia that may make them sleep. A gas bubble will be put in their eye to help it heal. Participants will receive immunosuppressive medications to avoid transplant rejection. Participants will be contacted yearly for up to 15 years.

Key facts

Study ID

NCT04339764

Run by

National Eye Institute (NEI)

People needed

20

Starts

2020-09-23

Expected to finish

2029-05-31

Last updated by the study team

2026-08-10

Who can join

Age: 55 and older, up to 95. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• A participant is not eligible if any of the following exclusion criteria are present:
• Participant is actively receiving another study medication / investigational product (IP).
• Participant has any condition that significantly increases risk of systemic corticosteroids or systemic steroid-sparing immuno-modulatory agents, such as uncontrolled diabetes mellitus (defined as HbA1C > 7.5 at Baseline), chronic hepatitis or liver failure (defined, as elevated liver function tests (LFTs): alanine transaminase (ALT) or aspartate transaminase (AST) >= 2x upper limit of normal at Baseline), kidney disease with an eGFR <25 mL/min/1.73 m2 for 3 months or more or by any other evidence of disease chronicity, or present infection with HIV, syphilis, tuberculosis, hepatitis B, or hepatitis C (past infection now resolved, where applicable, is not exclusionary; but persistent infection, even if latent, is exclusionary).
• Participant has diagnosis of a malignancy expected to affect two-year survival.
• Participant is pregnant, breast-feeding, or planning to become pregnant through the first 12 months of the study.
• Participant has a family history of a retinal degeneration other than AMD suspected to play a role in the ocular phenotype of the participant in the judgment of the investigator, based on disease features and mode of inheritance, such as in a case of autosomal dominant retinal degeneration in a parent or child.
• Participant is taking, or has taken within the previous year, medication with known potential toxicity to the retina, optic nerve, or lens (such as chloroquine, hydroxychloroquine, ethambutol).
• Participant is taking any form of systemic anticoagulation which cannot be stopped for an indefinite period of time without significant risk. The determination of significant risk to the participant must be at the discretion of the study investigators and prescribing physician (or qualified alternative).
• Participant is unable or unwilling to give informed consent that includes use of medical records and clinical samples for current and future research.
• Study Eye Exclusion Criteria:
• The study eye has macular, subretinal or choroidal neovascularization, as assessed by FA and OCT; or any history of such neovascularization (as assessed by past available records or images).
• The study eye has serous or hemorrhagic pigment epithelial detachment, as assessed by FA and OCT, that is clinically significant in the judgment of the investigator.
• The study eye has a history of photodynamic therapy (PDT) or macular thermal laser photocoagulation, or history of intravitreal injection of anti-vascular endothelial growth factor (VEGF) agents or corticosteroids (excepting medications used peri-operatively at prior cataract surgery). The study eye has had intravitreal injections (anti-complement therapy) for treatment of dry AMD in the previous 12 weeks before enrollment. Any intravitreal injections with anti-complement therapy prior to 12 weeks are not exclusionary.
• The study eye has an axial length > 25.0 mm.
• The study eye has had surgery in the previous 12 weeks, or laser capsulotomy in the previous four weeks.
• The study eye has chronic glaucoma; OR significant ocular hypertension, defined as documented intraocular pressure of >= 26 mmHg on at least two occasions in the absence of self-limited acute glaucoma; OR history of probable or definite steroid response manifesting as acute glaucoma or ocular hypertension, even if self-limited and no longer present; OR the fellow eye has evidence for present or past glaucoma or ocular hypertension judged to significantly impact the risk of glaucoma in the study eye (including history of probable or definite steroid response). (Note: History of self-limited acute glaucoma in a study or fellow eye, if not secondary to steroid response, and if now resolved and not expected to recur (e.g., history of elevated intraocular pressure from retained visco-elastic after cataract surgery), is not exclusionary. History of glaucoma or ocular hypertension in the fellow eye, if not felt to significantly impact risk of glaucoma in the study eye, is not exclusionary.)
• The study eye has a condition materially increasing the risks of surgery or potentially affecting visual function over the next two years in the judgment of the investigator, such as chronic uveitis, diabetic retinopathy, keratitis, scleritis, optic neuropathy, untreated retinal detachment, macular edema from prior vein occlusion or other cause, proliferative vitreoretinopathy (PVR), vitreous hemorrhage, pathologic myopia, etc. A history of such conditions is not exclusionary, if judged to not materially increase risks of surgery or to potentially affect vision in the next two years in the opinion of the investigator.

Where it is running

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (enrolling)
• Johns Hopkins University — Baltimore, Maryland, United States

Full record on ClinicalTrials.gov

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