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Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

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

A Phase I/IIa Trial for Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration

Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration (NCT04339764) is a Phase 1 / Phase 2 interventional studying Dry Age-Related Macular Degeneration and Geographic Atrophy, sponsored by National Eye Institute (NEI). RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Dry Age-Related Macular Degeneration, a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 20 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

* Who May Qualify: Participant Eligibility Criteria: To be eligible, the following inclusion criteria must be met, where applicable. - Participant must be 55 years of age or older. - Participant must have a diagnosis of dry AMD, defined as presence (or history, as documented in available color fundus photographs) of at least one medium or large druse (greater than or equal to 63 micrometer diameter) in the macula in at least one eye; AND presence of GA in at least one eye. - Participant must understand and sign the protocol s willing to sign a consent form document. - Any participant of childbearing potential must have a negative pregnancy test at screening and must be willing to undergo pregnancy testing prior to RPE transplantation. - Any participant of childbearing potential and any participant able to father children who has a partner of childbearing potential must have (or have a partner who has) had a hysterectomy or vasectomy, be completely abstinent from intercourse, or must agree to practice an effective method of contraception through Month 12 in the study. Acceptable methods of contraception include: - Hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring), - Intrauterine device, - Barrier methods (diaphragm, condom) with spermicide, or - Surgical sterilization (tubal ligation). - Participant must be medically able to comply with the study treatment (including ability to safely receive anesthesia for surgery), study testing and procedures, and follow-up visits. Study Eye/Fellow Eye Eligibility Criteria The participant must have at least one eye meeting all inclusion criteria and none of the exclusion criteria listed below. The fellow eye must also meet the relevant eligibility criteria listed below. Study Eye Who May Qualify: ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
* INCLUSION CRITERIA: Participant Eligibility Criteria: To be eligible, the following inclusion criteria must be met, where applicable. * Participant must be 55 years of age or older. * Participant must have a diagnosis of dry AMD, defined as presence (or history, as documented in available color fundus photographs) of at least one medium or large druse (greater than or equal to 63 micrometer diameter) in the macula in at least one eye; AND presence of GA in at least one eye. * Participant must understand and sign the protocol s informed consent document. * Any participant of childbearing potential must have a negative pregnancy test at screening and must be willing to undergo pregnancy testing prior to RPE transplantation. * Any participant of childbearing potential and any participant able to father children who has a partner of childbearing potential must have (or have a partner who has) had a hysterectomy or vasectomy, be completely abstinent from intercourse, or must agree to practice an effective method of contraception through Month 12 in the study. Acceptable methods of contraception include: * Hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring), * Intrauterine device, * Barrier methods (diaphragm, condom) with spermicide, or * Surgical sterilization (tubal ligation). * Participant must be medically able to comply with the study treatment (including ability to safely receive anesthesia for surgery), study testing and procedures, and follow-up visits. Study Eye/Fellow Eye Eligibility Criteria The participant must have at least one eye meeting all inclusion criteria and none of the exclusion criteria listed below. The fellow eye must also meet the relevant eligibility criteria listed below. Study Eye Inclusion Criteria: * The study eye must have one or more regions of geographic atrophy with total area of 1 disc area or more. A region of geographic atrophy is defined as an area of uniform hypofluorescence on fundus autofluorescence (FAF) imaging, with greatest linear dimension at least 500 micrometer, with a border within 500 micrometer of the foveal center, not compatible with pigmentary changes, drusen, RPE detachment, drusenoid RPE detachment, hemorrhage, or other lesion. (Note: If macular geographic atrophy is contiguous with peripapillary atrophy, complicating calculation of total area, only atrophy temporal to a vertical line placed a half disc diameter temporal to the temporal border of the disc will be included in the total area of geographic atrophy calculated for eligibility purposes.) * For participants in the first cohort, the study eye must have an ETDRS best-corrected visual acuity (BCVA) letter score of \<= 53 and \>= CF (i.e., Snellen equivalent between 20/100 and CF), and the fellow eye must have a letter score no more than five letters worse than the study eye using Electronic Visual Acuity (EVA) testing. If the study eye is CF vision, then the fellow eye must be both (1) CF or better vision and (2) subjectively as good or better than the study eye according to the subject s perception. (Note: Letter scores within five or fewer letters of each other are accordingly considered equal for eligibility determination, and other factors may be used to select the study eye if both are eligible by BCVA. * For participants in the second cohort, the study eye must have an ETDRS best-corrected visual acuity (BCVA) letter score of \<= 58 and \>= CF (i.e., Snellen equivalent between 20/80 and CF), and the fellow eye must have a letter score no more than five letters worse than the study eye using Electronic Visual Acuity (EVA) testing. If the study eye is CF vision, then the fellow eye must be both (1) CF or better vision and (2) subjectively as good or better than the study eye according to the subject s perception. (Note: Letter scores within five or fewer letters of each other are accordingly considered equal for eligibility determination, and other factors may be used to select the study eye if both are eligible by BCVA. * The compromise in visual acuity for the study eye must be judged predominantly secondary to dry AMD, in the judgment of the investigator. * The study eye must have clarity of ocular media and degree of pupil dilation sufficient to permit adequate fundus photography and safe vitrectomy surgery. * The study eye must be either pseudophakic or aphakic. EXCLUSION CRITERIA: 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, chronic hepatitis or liver failure, chronic renal failure, 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.

Treatments Being Tested

DRUG

iPSC-derived RPE/PLGA transplantation

iPSC-derived RPE/PLGA transplantation

Locations (2)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Johns Hopkins University
Baltimore, Maryland, United States
National Institutes of Health Clinical Center
Bethesda, Maryland, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT04339764), the sponsor (National Eye Institute (NEI)), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT04339764 clinical trial studying?

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: Particip… The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT04339764?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT04339764?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT04339764. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT04339764. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.