Dry Age-Related Macular DegenerationPhase 1/2NCT04339764
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
A Phase 1/2 study testing iPSC-derived RPE/PLGA transplantation in people with Dry Age-Related Macular Degeneration and Geographic Atrophy, run by National Eye Institute (NEI) at 2 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 55 to 95 years
- Conditions the study is for
- Dry Age-Related Macular Degeneration, Geographic Atrophy
- What is being tested?
- iPSC-derived RPE/PLGA transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 2 sitesUnited States
- Study dates
- 23 Sep 2020to 31 May 2029
- 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 55 to 95 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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 > 26.0 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Not yet recruiting
Johns Hopkins University
Baltimore, Maryland, 21205
Mais Hamdawi, 410-343-9434, malhamd2@jh.edu
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR), 800-411-1222 ext. TTY8664111010, prpl@cc.nih.gov
About this study
Official title: 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
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.
Read more
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.
Read the detailed description
Age-related macular degeneration (AMD) is a leading cause of vision loss among the elderly. There are limited treatment options for geographic atrophy (GA), the advanced stage of dry AMD, in which cells of the neurosensory retina and associated retinal pigment epithelium (RPE) gradually degenerate and die. Advances in stem cell biology allowing differentiation of pluripotent cells into RPE in vitro make feasible a cell-based strategy for potential treatment of AMD, and recent methods for induced pluripotent stem cell (iPSC) generation offer promise of individualized autologous therapy. Such an approach involves generation of iPSC from somatic cells taken from a patient with GA, differentiation of iPSC into RPE grown as a monolayer on a thin scaffold in vitro, and transplantation of the RPE/scaffold construct into a small region in the subretinal space of the same patient, with a goal of rescuing the overlying neurosensory retina from further degeneration.
Objective: To evaluate the safety and feasibility of subretinal transplantation of iPSC-derived RPE, grown as a monolayer on a biodegradable poly lactic-co-glycolic acid (PLGA) scaffold, as a potential autologous cell-based therapy for GA associated with AMD.
Study Population: Five participants will undergo RPE transplantation in one eye. Eligible eyes will have GA, best-corrected visual acuity (BCVA) between 20/100 and inclusive of counting fingers (CF), and a fellow eye that has same or better BCVA. If the National Eye Institute (NEI) Data and Safety Monitoring Committee (DSMC) gives clearance to proceed based on review of data from the first cohort, a second cohort of up to seven additional participants with GA, BCVA between 20/80 and CF (inclusive) in the eye being considered for RPE transplantation, and same or better visual acuity in the other eye may undergo the procedure to gather additional safety and potential efficacy data useful for planning future studies. Up to 20 participants may be enrolled to allow for screening failures, for participants withdrawing from the study prior to RPE transplantation, or cases where the RPE cell transplantation does not occur due to intraoperative surgical considerations.
Design: In this phase I/IIa, prospective, single-arm, multi-center clinical trial, participants will undergo subretinal transplantation of autologous iPSC-derived RPE in one eye and will be followed for five years after surgery.
Outcome Measures: The primary outcome measure is the safety of RPE/PLGA transplantation, as determined by assessment of visual acuity change and summary of adverse events at 12 months after RPE/PLGA transplantation. Secondary outcome measures include visual acuity change and adverse event reporting at 24 and 60 months, and changes in the following at 12, 24 and 60 months as compared with baseline, assessed in the transplanted region, and compared where applicable with other areas in the macula, and/or with corresponding regions in the fellow eye: retinal sensitivity and fixation parameters assessed by microperimetry; multifocal electroretinography (mfERG) responses; macular structure on cross-sectional and en face imaging by optical coherence tomography (OCT); macular features on color, single-wavelength reflectance, and fundus autofluorescence (FAF) photography; and fluorescein angiography (FA). Some NEI and JHU participants may undergo imaging of photoreceptor/RPE features using adaptive-optics-assisted macular imaging under a separate NEI protocol (e.g., 15-EI-0020).
- Being tested
- iPSC-derived RPE/PLGA transplantation
- Keywords
- Retina, Cell Therapy, Vitrectomy
- Medical subject headings
- Geographic Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
iPSC-derived RPE/PLGA transplantationDrug
iPSC-derived RPE/PLGA transplantation
| Study group | Role | What they receive |
|---|---|---|
| Participants receiving intervention | Experimental | Participants receiving intervention Receives: Drug: iPSC-derived RPE/PLGA transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Summary of adverse events
safety measure
Measured: 12 months compared to final Baseline prior to surgery
Visual acuity change
safety measure
Measured: 12 months compared to final Baseline prior to surgery
Secondary outcomes (7)
Retinal structure (fluorescein angiography)
Safety and efficacy measure
Measured: 12, 24, and 60 month
Retinal Structure (color and autofluorescence imaging)
Safety and efficacy measure
Measured: 12, 24, and 60 months
Multifocal electroretinography responses
Safety and efficacy measure
Measured: 12, 24, and 60 months
Retinal sensitivity and fixation (microperimetry)
Safety and efficacy measure
Measured: 12, 24, and 60 months
Retinal Structure (optical coherence tomography)
Safety and efficacy measure
Measured: 12, 24, and 60 months
Visual acuity change
Safety and efficacy measure
Measured: 24 and 60 months
Summary of adverse events
Safety and efficacy measure
Measured: 24 and 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2020
- Study start23 Sep 2020
- Primary completion31 May 2029 (estimated)
- Study completion31 May 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dry Age-Related Macular DegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Eye Institute (NEI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Emily Y Chew, M.D., Principal investigator, National Eye Institute (NEI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04339764
- Sponsor's protocol ID
- 200052
- Other IDs
- 20-EI-0052
- Status last verified
- 6 Aug 2026
- First submitted
- 8 Apr 2020
- Last updated
- 5 Nov 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04339764
- Acronym / sponsor ID
- 200052
- Keywords
- Retina, Cell Therapy, Vitrectomy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- National Eye Institute (NEI)US National Institutes of Health
- Study start
- 23 Sep 2020 (actual)
- Primary completion
- 31 May 2029 (estimated)
- Study completion
- 31 May 2029 (estimated)
- First posted
- 9 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Age-related macular degeneration (AMD) is a leading cause of vision loss among the elderly. There are limited treatment options for geographic atrophy (GA), the advanced stage of dry AMD, in which cells of the neurosensory retina and associated retinal pigment epithelium (RPE) gradually degenerate and die. Advances in stem cell biology allowing differentiation of pluripotent cells into RPE in vitro make feasible a cell-based strategy for potential treatment of AMD, and recent methods for induced pluripotent stem cell (iPSC) generation offer promise of individualized autologous therapy. Such an approach involves generation of iPSC from somatic cells taken from a patient with GA, differentiation of iPSC into RPE grown as a monolayer on a thin scaffold in vitro, and transplantation of the RPE/scaffold construct into a small region in the subretinal space of the same patient, with a goal of rescuing the overlying neurosensory retina from further degeneration.
Objective: To evaluate the safety and feasibility of subretinal transplantation of iPSC-derived RPE, grown as a monolayer on a biodegradable poly lactic-co-glycolic acid (PLGA) scaffold, as a potential autologous cell-based therapy for GA associated with AMD.
Study Population: Five participants will undergo RPE transplantation in one eye. Eligible eyes will have GA, best-corrected visual acuity (BCVA) between 20/100 and inclusive of counting fingers (CF), and a fellow eye that has same or better BCVA. If the National Eye Institute (NEI) Data and Safety Monitoring Committee (DSMC) gives clearance to proceed based on review of data from the first cohort, a second cohort of up to seven additional participants with GA, BCVA between 20/80 and CF (inclusive) in the eye being considered for RPE transplantation, and same or better visual acuity in the other eye may undergo the procedure to gather additional safety and potential efficacy data useful for planning future studies. Up to 20 participants may be enrolled to allow for screening failures, for participants withdrawing from the study prior to RPE transplantation, or cases where the RPE cell transplantation does not occur due to intraoperative surgical considerations.
Design: In this phase I/IIa, prospective, single-arm, multi-center clinical trial, participants will undergo subretinal transplantation of autologous iPSC-derived RPE in one eye and will be followed for five years after surgery.
Outcome Measures: The primary outcome measure is the safety of RPE/PLGA transplantation, as determined by assessment of visual acuity change and summary of adverse events at 12 months after RPE/PLGA transplantation. Secondary outcome measures include visual acuity change and adverse event reporting at 24 and 60 months, and changes in the following at 12, 24 and 60 months as compared with baseline, assessed in the transplanted region, and compared where applicable with other areas in the macula, and/or with corresponding regions in the fellow eye: retinal sensitivity and fixation parameters assessed by microperimetry; multifocal electroretinography (mfERG) responses; macular structure on cross-sectional and en face imaging by optical coherence tomography (OCT); macular features on color, single-wavelength reflectance, and fundus autofluorescence (FAF) photography; and fluorescein angiography (FA). Some NEI and JHU participants may undergo imaging of photoreceptor/RPE features using adaptive-optics-assisted macular imaging under a separate NEI protocol (e.g., 15-EI-0020).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants receiving intervention | Experimental | Drug: iPSC-derived RPE/PLGA transplantationParticipants receiving intervention |
Who may qualify
- Ages
- 55 to 95 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- 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.
Key exclusion criteria (17 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| iPSC-derived RPE/PLGA transplantationiPSC-derived RPE/PLGA transplantation | Drug | Participants receiving intervention |
Primary outcome
Summary of adverse events
Time frame: 12 months compared to final Baseline prior to surgery
safety measure
Visual acuity change
Time frame: 12 months compared to final Baseline prior to surgery
safety measure
7 secondary outcomes
Retinal structure (fluorescein angiography)
Time frame: 12, 24, and 60 month
Retinal Structure (color and autofluorescence imaging)
Time frame: 12, 24, and 60 months
Multifocal electroretinography responses
Time frame: 12, 24, and 60 months
Retinal sensitivity and fixation (microperimetry)
Time frame: 12, 24, and 60 months
Retinal Structure (optical coherence tomography)
Time frame: 12, 24, and 60 months
Visual acuity change
Time frame: 24 and 60 months
Summary of adverse events
Time frame: 24 and 60 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Emily Y Chew, M.D. principal investigator
National Eye Institute (NEI)
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing iPSC-derived RPE/PLGA transplantation in people with Dry Age-Related Macular Degeneration and Geographic Atrophy, run by National Eye Institute (NEI) at 2 sites in United States.
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 21 inclusion and 17 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 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. 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 Ellaine M Galindez-Balut ((301) 402-4726, ellaine.galindez-balut@nih.gov), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Summary of adverse events (time frame: 12 months compared to final Baseline prior to surgery). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 Nov 2026.