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

NCT04339764 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: 20005220-EI-0052
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Dry Age-Related Macular Degeneration; Geographic Atrophy

Keywords

Retina; Cell Therapy; Vitrectomy

Outcome Measures

Primary Outcomes (2)

• Summary of adverse events

  safety measure

  12 months compared to final Baseline prior to surgery

• Visual acuity change

  safety measure

  12 months compared to final Baseline prior to surgery

Secondary Outcomes (7)

• Retinal structure (fluorescein angiography)

  12, 24, and 60 month

• Retinal Structure (color and autofluorescence imaging)

  12, 24, and 60 months

• Multifocal electroretinography responses

  12, 24, and 60 months

• Retinal sensitivity and fixation (microperimetry)

  12, 24, and 60 months

• Retinal Structure (optical coherence tomography)

  12, 24, and 60 months

Study Arms (1)

Participants receiving intervention

EXPERIMENTAL

Participants receiving intervention

Drug: iPSC-derived RPE/PLGA transplantation

Interventions

iPSC-derived RPE/PLGA transplantation DRUG

iPSC-derived RPE/PLGA transplantation

Participants receiving intervention

Eligibility Criteria

• Age: 55 Years - 95 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant Eligibility Criteria:
• 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

You may not qualify if:

• 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.
• Participant is actively receiving another study medication / investigational product (IP).
• 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 retina, optic nerve, or lens (such as chloroquine, hydroxychloroquine, ethambutol).
• Participant is taking any form of systemic anticoagulation which cannot be stopped for an indefinite period of time without significant risk. The determination of significant risk to the participant must be at the discretion of the study investigators and prescribing physician (or qualified alternative).
• Participant is unable or unwilling to give informed consent that includes use of medical records and clinical samples for current and future research.
• The study eye has macular, subretinal or choroidal neovascularization, as assessed by FA and OCT; or any history of such neovascularization (as assessed by past available records or images).
• The study eye has serous or hemorrhagic pigment epithelial detachment, as assessed by FA and OCT, that is clinically significant in the judgment of the investigator.

Sponsors & Collaborators

• National Eye Institute (NEI): lead

Study Sites (2)

Johns Hopkins University

Baltimore, Maryland, 21205, United States

NOT YET RECRUITING

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Geographic Atrophy

Condition Hierarchy (Ancestors)

Macular Degeneration; Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Officials

• Emily Y Chew, M.D.

  National Eye Institute (NEI)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ellaine M Galindez-Balut

CONTACT

(301) 402-4726; ellaine.galindez-balut@nih.gov

Emily Y Chew, M.D.

CONTACT

(301) 496-6583; echew@nei.nih.gov

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 8, 2020
• First Posted: April 9, 2020
• Study Start: September 23, 2020
• Primary Completion (Estimated): May 31, 2029
• Study Completion (Estimated): May 31, 2029
• Last Updated: February 5, 2026

Record last verified: 2026-01-29

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)