Actively Recruiting
A Phase I/IIa Trial of Autologous Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium Transplantation for Geographic Atrophy in Dry Age-Related Macular Degeneration
Led by National Eye Institute (NEI) · Updated on 2026-02-05
20
Participants Needed
2
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying a new approach for treating geographic atrophy, an advanced form of dry age-related macular degeneration (AMD) that causes vision loss in people over 50. The trial aims to test the safety of replacing damaged retinal pigment epithelium (RPE) cells with younger cells derived from the participant's own induced pluripotent stem cells (iPSC). This phase I/IIa study evaluates whether transplanting these cells can be a potential future treatment for this eye condition. Participants will undergo surgery to receive a transplant of iPSC-derived RPE cells grown on a biodegradable scaffold placed under the retina of one eye. The cells are made from the participant's blood cells, converted in the lab. During surgery, participants will receive anesthesia and a gas bubble will be inserted into the eye to aid healing. After surgery, immunosuppressive medications will be given to reduce the risk of transplant rejection. The study includes up to two cohorts with slightly different vision criteria, and up to 20 participants may be enrolled to allow for screening and surgical considerations. Participants will be closely monitored through at least 14 study visits over five and a half years, including eye exams, imaging tests like fluorescein angiography and optical coherence tomography, and electrical tests of eye function. Safety and vision changes will be tracked at 12, 24, and 60 months after transplantation. Researchers will also follow participants yearly for up to 15 years to gather long-term data. The main focus is on safety outcomes and changes in visual acuity compared to baseline before surgery.
CONDITIONS
Brief Title
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant is 55 years of age or older.
- Diagnosis of dry age-related macular degeneration with geographic atrophy in at least one eye.
- Understanding and signing of the informed consent document.
- Negative pregnancy test for participants of childbearing potential.
- Use of effective contraception through Month 12 for participants or partners of childbearing potential.
- Medically able to comply with study treatment, procedures, and follow-up visits.
- Study eye must have geographic atrophy with specified size and location criteria.
- Study eye with best-corrected visual acuity between counting fingers and 20/100 or 20/80 depending on cohort.
- Fellow eye with same or better visual acuity than the study eye.
- Study eye must be pseudophakic or aphakic with clear ocular media and adequate pupil dilation.
You will not qualify if you...
- Currently receiving another investigational product.
- Conditions increasing risks of systemic corticosteroids or immunomodulatory agents, including uncontrolled diabetes, liver or kidney failure, or active infections like HIV or tuberculosis.
- Malignancy expected to affect two-year survival.
- Pregnancy, breastfeeding, or planning pregnancy through first 12 months.
- Family history of retinal degeneration other than AMD suspected to affect participant.
- Use of medications with known retinal toxicity within past year.
- Use of systemic anticoagulation that cannot be stopped safely.
- Unable or unwilling to give informed consent.
- Study eye with active or history of neovascularization, significant pigment epithelial detachment, or recent eye surgeries.
- Chronic glaucoma or significant ocular hypertension affecting study eye.
- Other eye conditions increasing surgical risks or affecting vision within two years as judged by investigator.
- Study eye axial length greater than 25.0 mm.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single transplantation procedure with follow-up
Participants undergo subretinal transplantation of autologous iPSC-derived retinal pigment epithelium (RPE) grown on a biodegradable scaffold to potentially treat geographic atrophy associated with age-related macular degeneration.
1 surgical visit and multiple follow-up visits
Duration - Up to 5 years after surgery
Participants are monitored for safety and visual function changes after transplantation, including assessments of visual acuity, retinal structure, and retinal sensitivity.
Regular visits for assessments at 12, 24, and 60 months post-transplantation
Trial Site Locations
Total: 2 locations
1
Johns Hopkins University
Baltimore, Maryland, United States, 21205
Not Yet Recruiting
2
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
E
Ellaine M Galindez-Balut
E
Emily Y Chew, M.D.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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