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

RecruitingPhase 1, Phase 2Ages 55 Years - 95 YearsAllNational Eye Institute (NEI)
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In plain words

What the trial is, and whether it could suit you

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. * Electroret

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
* INCLUSION CRITERIA: 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. * 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. EXCLUSION CRITERIA: 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. * Participant is taking, or has taken within the previous year, medication with known potential toxicity to the reti
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What it tests

  • iPSC-derived RPE/PLGA transplantation
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Where

2 sites
Johns Hopkins University
Baltimore, United States
National Institutes of Health Clinical Center
Bethesda, United States
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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