Actively Recruiting

Phase 1
Phase 2
Age: 55Years - 95Years
All Genders
NCT04339764

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

Led by National Eye Institute (NEI) · Updated on 2026-02-05

20

Participants Needed

2

Research Sites

453 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 55Years - 95Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 55 years or older
  • Diagnosed with dry age-related macular degeneration and geographic atrophy in at least one eye
  • Ability to understand and sign informed consent
  • Negative pregnancy test if of childbearing potential
  • Use of effective contraception through Month 12 if of childbearing potential or partner is
  • Medically able to safely receive anesthesia and comply with study procedures
  • Study eye has geographic atrophy area of 1 disc area or more near the foveal center
  • Study eye visual acuity between 20/100 and counting fingers (CF), with fellow eye vision similar or better
  • Study eye clarity and pupil dilation sufficient for photography and surgery
  • Study eye is pseudophakic or aphakic
Not Eligible

You will not qualify if you...

  • Currently receiving another investigational medication or product
  • Conditions increasing risk of corticosteroids or immunomodulatory agents (e.g., uncontrolled diabetes, chronic infections like HIV, hepatitis, tuberculosis)
  • Malignancy expected to affect two-year survival
  • Pregnancy, breastfeeding, or planning pregnancy through first 12 months
  • Family history suggesting non-AMD retinal degeneration
  • Use of medications toxic to retina or optic nerve in past year
  • Use of systemic anticoagulation that cannot be safely stopped
  • Unable or unwilling to provide informed consent
  • Study eye has neovascularization, significant pigment epithelial detachment, or prior certain eye treatments
  • Study eye axial length over 25.0 mm
  • Recent eye surgery or laser capsulotomy
  • Chronic glaucoma or significant ocular hypertension
  • Eye conditions increasing surgical risks or affecting vision over next two years (e.g., uveitis, diabetic retinopathy, macular edema)

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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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

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Research Team

E

Ellaine M Galindez-Balut

CONTACT

E

Emily Y Chew, M.D.

CONTACT

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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