RecruitingPhase 1Phase 2NCT04339764

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


Sponsor

National Eye Institute (NEI)

Enrollment

20 participants

Start Date

Sep 23, 2020

Study Type

INTERVENTIONAL

Conditions

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. 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.


Eligibility

Min Age: 55 YearsMax Age: 95 Years

Plain Language Summary

Simplified for easier understanding

This study tests an experimental treatment for geographic atrophy (GA) — an advanced form of dry age-related macular degeneration (AMD) that causes progressive, irreversible vision loss as the light-sensing cells of the retina die off. The treatment involves taking a patient's own skin cells, reprogramming them into stem cells (iPSCs), and then converting those stem cells into retinal pigment epithelium (RPE) cells — the supporting layer underneath the retina that is lost in GA. These laboratory-grown RPE cells are then surgically transplanted into the patient's own eye. Because the cells are made from the patient themselves, there is no risk of immune rejection. The study assesses whether this approach is safe and whether transplanted cells survive and slow the progression of vision loss. You may be eligible if: - You are 55 years of age or older - You have a diagnosis of dry AMD with geographic atrophy in at least one eye - Your vision in the study eye meets specific criteria (reduced but not completely absent) - You are medically able to undergo eye surgery under anaesthesia You may NOT be eligible if: - You have uncontrolled diabetes, active HIV, syphilis, tuberculosis, or hepatitis - You have a cancer diagnosis expected to affect your 2-year survival - You are pregnant, breastfeeding, or planning pregnancy in the first 12 months of the study - You are taking anticoagulants that cannot be safely stopped - You have a family history of a different inherited retinal disease - Your study eye has signs of wet AMD (abnormal blood vessels growing under the retina) Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGiPSC-derived RPE/PLGA transplantation

iPSC-derived RPE/PLGA transplantation


Locations(2)

Johns Hopkins University

Baltimore, Maryland, United States

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

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NCT04339764


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