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Clinical Trial Highlight: Stem Cell Transplants for Dry AMD

Clinical Trial Summary

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

In this investigational, first-in-human, phase I/IIa, single-arm clinical trial, participants will undergo subretinal transplantation of autologous induced pluripotent stem cell-derived retinal pigment epithelium (iRPE) in one eye and will be followed for five years after surgery. The iRPE patch consists of an approximately 2 X 4 mm oval sheet in which cells are grown on a biodegradable PLGA scaffold material.

The goal is to transplant the iRPE patch into an area alongside a border of geographic atrophy causing central vision loss. Five participants meeting eligibility criteria, including best-corrected visual acuity between 20/100 and 20/500 in the worse-seeing eye being considered for the study, will be enrolled initially.

If adequate safety is demonstrated among these participants, up to an additional seven eligible participants with best-corrected visual acuity between 20/80 and 20/500 in the worse-seeing eye will be enrolled.  The primary outcome is the safety of iRPE transplantation.  Secondary outcomes will include measures of the durability and effects of the iRPE patch.


Age-related macular degeneration (AMD) is a leading cause of vision loss among people 55 years old and older. There is no treatment for geographic atrophy (GA), the advanced stage of dry AMD, in which cells of the retina and associated retinal pigment epithelium (RPE) gradually degenerate and die.

One approach being explored to prevent worsening of dry macular degeneration is to place new RPE cells under the retina to better support photoreceptors, the light-sensing cells of the retina.  Early efforts have been limited by the ability to effectively transplant cells under the retina and to avoid rejection of cells by the immune system.

Research advances have made it possible to re-program adult cells, like cells from the blood, into stem cells. These can then be used to create new RPE cells, custom-made for an individual, offering the potential of personalized medicine.

This trial will test whether RPE made in this way, called autologous induced pluripotent stem cell-derived RPE (iPSCs), can be feasibly and safely transplanted under the retina as a potential treatment for advanced dry macular degeneration.


This clinical trial will evaluate the safety and feasibility of investigational subretinal transplantation of RPE made from induced pluripotent stem cells as a potential autologous cell-based therapy for advanced dry AMD.

Who is eligible to participate?

The National Eye Institute is looking for up to twelve (12) participants to undergo RPE transplantation in one eye. Some of the key eligibility criteria include: 

  • Age: 55 years of age or older;
  • Diagnosis of AMD and presence of geographic atrophy (GA) involving the central retina in at least one eye;
  • Best-corrected visual acuity (BCVA) between 20/100 and 20/500 (first cohort), or between 20/80 and 20/500 (second cohort), and a fellow eye that has same or better BCVA;
  • No history of exudative (wet) AMD or prior treatment for exudative AMD in EITHER eye;
  • No glaucoma or significant elevation of intraocular pressure;
  • Medical status permitting safe surgery
  • Not currently receiving intervention in another study

How to Find More Information

More information about this study is available at the clinical trials registry,  

Information about this study and other studies at the NIH and elsewhere is also available in the Resources section. 

How to be Considered for the Study or to Refer a Patient

Individuals interested in being considered for this study are encouraged to discuss potential participation with their eye care provider and to request referral.

Providers can make a referral by contacting Ellaine Galindez-Ballut, NEI Recruitment Coordinator at

Frequently Asked Questions

Can I be considered for the study without referral from a medical provider?
Yes.  You can contact Ellaine Galindez-Ballut, NEI Recruitment Coordinator at However, we highly encourage submission of an eye provider’s referral note and/or recent clinic note, so that we may offer advice about whether scheduling a pre-screening or screening visit at the NIH could be worthwhile.

Can I enroll in the study if I’m younger than 55 years of age?
This clinical trial only allows participants 55 years of age or older. This is because geographic atrophy in younger individuals often happens because of other types of macular degeneration, not age-related macular degeneration, and this study is not designed to evaluate other conditions.

I have other eye conditions besides dry age-related macular degeneration. Am I eligible?
This will depend on the pre-existing condition. For example, patients with a mild cataract or an intraocular lens implant placed at prior cataract surgery can be considered, while those with any history of wet macular degeneration or chronic glaucoma are not eligible.

We encourage interested individuals to discuss the study with their eye care providers, and to request a referral that includes information about eye and health history so that we can offer preliminary advice about potential eligibility prior to consideration of a visit to the NIH.

How am I going to be screened for the study?
If you or a referring provider sends information about your eye or health history as part of a referral, we can review these documents to provide preliminary advice about potential eligibility prior to consideration of a visit to the NIH.

Potential participants who visit the NIH will undergo evaluation in the NEI Eye Clinic to determine whether they meet the eligibility criteria.  Eye evaluation includes visual acuity measurement, a dilated eye exam, imaging of the retina, a fluorescein angiogram, and other tests of eye structure and function.  Medical evaluation includes a history and physical, bloodwork, urinalysis, a chest X ray, EKG, and additional testing.  The full screening process is usually completed over more than one day.

Is my vision going to improve with this procedure?
This is a first-in-human clinical trial testing feasibility and safety.  Placement of the iRPE patch is not expected to benefit participants’ vision in a manner that would aid reading or other fine discriminatory tasks, given the already-advanced state of vision loss of the eye.  The study will lead to knowledge that will enhance understanding of autologous stem cell-based therapy for potential treatment of GA associated with AMD.

How am I going to know if there’s a problem?
All surgeries carry risks. Those will be explained to you by the clinical trial investigators. Participants will be monitored during all study visits for adverse events by the clinical trial investigators. Participants will be instructed to contact a clinical trial investigator at any time if they experience any new concerns. 

Will my other eye undergo the procedure?
For safety reasons, this trial will only involve surgery for the worse-seeing eye.  The other eye will not receive the iRPE patch.

How long will I be in this study?
If you enroll and are able to undergo the procedure, you will be followed in the study for approximately five and a half years.  The iRPE patch takes around 6 months to make, and participants are followed for five years after the procedure.  You have the right to withdraw from the study at any time.

Will participation in this study affect my eligibility for other studies in the future?
This depends on the study being considered in the future.  Many studies do not allow for participation if a person is currently enrolled in another study.  And some studies may exclude people who have had certain interventions done in earlier studies.

How does this trial compare with other studies testing cell-based therapies for macular degeneration?
This study will enroll people with advanced dry age-related macular degeneration, but not other kinds of macular degeneration, and not wet macular degeneration.  

The goal is tissue replacement, specifically the replacement of RPE cells, a supportive layer of cells underneath the retina.  RPE cells in this study are manufactured from stem cells made from an individual’s own blood cells, which offers the potential of a personalized therapy and may decrease the chance that the body would reject the cells.  RPE cells in this study are pre-formed in a solid layer and delivered inside the eye as a patch ready for normal function, instead of being injected inside the eye as a mixture of individual cells.

How will I visit the NIH, and will there be any compensation for the study?
We recognize that those considered for this study have impaired vision and many may have impaired mobility that may make travel to NIH challenging.  For participants who enroll, this study offers reimbursement for, or payment of, travel to and from the NIH within the United States, lodging, and meals.  We do not offer any additional payment or compensation for participation. Those interested in visiting the NIH for consideration of participation may be offered assistance on a case-by-case basis.


Below is information you might find useful as you consider being part of this study and whether you might be potentially eligible to participate.  For questions or detailed materials, please contact: Ellaine Galindez-Ballut, NEI Recruitment Coordinator at

NIH launches first U.S. clinical trial of patient-derived stem cell therapy to replace and repair dying cells in retina

NEI’s Ocular and Stem Cell Translational Research

NIH researchers rescue photoreceptors, prevent blindness in animal models of retinal degeneration

Information about this study at the registry:

Information about NEI Clinical Trials:

Information about NEI’s Eye Clinic: 

Information about other clinical trials:   

Last updated: March 1, 2022