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Protocol Details

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

This study is currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number

20-EI-0052

Sponsoring Institute

National Eye Institute (NEI)

Recruitment Detail

Type: Participants currently recruited/enrolled
Gender: Male & Female
Min Age: 55
Max Age: N/A

Referral Letter Required

No

Population Exclusion(s)

Pregnant Women;
Adults who are or may become unable to consent;
Children

Special Instructions

Currently Not Provided

Keywords

Retina;
Cell Therapy;
Vitrectomy

Recruitment Keyword(s)

None

Condition(s)

Age-Related Macular Degeneration

Investigational Drug(s)

Autologous iPSC-derived RPE on PGLA

Investigational Device(s)

None

Intervention(s)

Device: iPSC-derived RPE/PLGA transplantation
Biological/Vaccine: Atuologous iPSC-derived RPE on PGLA

Supporting Site

National Eye Institute

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 either 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 be contacted yearly for up to 15 years.

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Eligibility

INCLUSION 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 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 must have (or have a partner who has) had a hysterectomy or vasectomy, be completely abstinent from intercourse, or must agree to practice two effective methods 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 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 less than or equal to 53 and greater than or equal to 14 (i.e., Snellen equivalent between 20/100 and 20/500), 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. (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 less than or equal to 58 and greater than or equal to 14 (i.e., Snellen equivalent between 20/80 and 20/500), 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. (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.

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, chronic hepatitis or liver failure, chronic renal failure, 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 retina, optic nerve, or lens (such as chloroquine, hydroxychloroquine, ethambutol).

-Participant is unable or unwilling to give informed consent that includes use of medical records and clinical samples for current and future research.

Study/Fellow Eye Exclusion Criteria:

-The study eye AND the fellow eye must not have 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 AND the fellow eye must not have any serous or hemorrhagic pigment epithelial detachment, as assessed by FA and OCT.

-The study eye must not have a cataract dense enough to preclude visualization adequate for vitreoretinal surgery, in the judgment of the investigator.

-The study eye AND the fellow eye must not have any history of photodynamic therapy (PDT) or macular thermal laser photocoagulation, or history of intravitreal injection of anti-vascular endothelial growth factor (VEGF) agents or corticosteroids (excepting medications used peri-operatively at prior cataract surgery).

-The study eye must not have an axial length > 25.0 mm.

-The study eye must not have had any surgery in the previous 12 weeks, or laser capsulotomy in the previous four weeks.

-The study eye must not have chronic glaucoma; OR significant ocular hypertension, defined as documented intraocular pressure of greater than or equal to 26 mmHg on at least two occasions in the absence of self-limited acute glaucoma; OR history of probable or definite steroid response manifesting as acute glaucoma or ocular hypertension, even if self-limited and no longer present; and the fellow eye must not have evidence for present or past glaucoma or ocular hypertension judged to significantly impact the risk of glaucoma in the study eye (including history of probable or definite steroid response). (Note: History of self-limited acute glaucoma in a study or fellow eye, if not secondary to steroid response, and if now resolved and not expected to recur (e.g., history of elevated intraocular pressure from retained visco-elastic after cataract surgery), is not exclusionary. History of glaucoma or ocular hypertension in the fellow eye, if not felt to significantly impact risk of glaucoma in the study eye, is not exclusionary.)

-The study eye must not have a condition materially increasing the risks of surgery or potentially affecting visual function over the next two years in the judgment of the investigator, such as chronic uveitis, diabetic retinopathy, keratitis, scleritis, optic neuropathy, untreated retinal detachment, macular edema from prior vein occlusion or other cause, proliferative vitreoretinopathy (PVR), vitreous hemorrhage, pathologic myopia, etc. A history of such conditions is not exclusionary, if judged to not materially increase risks of surgery or to potentially affect vision in the next two years in the opinion of the investigator.


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Citations:

Not Provided

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Contacts:

Principal Investigator

Referral Contact

For more information:

Henry E. Wiley IV, M.D.
National Eye Institute (NEI)



Angel H. Garced, R.N.
National Eye Institute (NEI)
National Institutes of Health
Building 10
Room 10D45
10 Center Drive
Bethesda, Maryland 20892
(301) 594-3141
garceda@nei.nih.gov

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: 1-866-411-1010
PRPL@cc.nih.gov

Clinical Trials Number:

NCT04339764

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