This project intends to perform autologous transplantation of induced pluripotent stem cell-derived retinal pigment epithelium (iPSC-RPE). The clinical-grade RPE will be transplanted into subretinal space to treat refractory age-related macular degeneration. The efficacy and safety of RPE transplants to treat macular degeneration will be monitored and analyzed with results from EDTRS, BCVA, OCT, ERG, microperimetry, and fluorescein angiography, before and after the treatment.
The investigators will generate iPSC lines from recruited participants and differentiate the iPSC into RPE. Autologous iPSC-derived RPE will be transplanted into participants eyes by subretinal injections. The safety and efficacy will be closely monitored and analyzed.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Autologous transplantation of iPSC-derived RPE
Beijing Tongren Hospitol,Capital Medical University
Beijing, Beijing Municipality, China
RECRUITINGSafety measure
Safety will be assessed by Adverse Events (AEs) of special interest in regards to the investigational product. This will include obtaining information about Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, any AE that causes the subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer (irrespective of prior history), ectopic or proliferative cell growth (RPE or non-RPE) with adverse clinical consequence, unexpected, clinically significant AE possibly related to the cell transplant procedure or the investigational product (autologous iPSC-RPE), pregnancy in a female subject or the partner of a male subject and pregnancy outcome.
Time frame: 12 months
Best Corrected Visual Acuity (BCVA)
Change in visual acuity will be measured by Early Treatment Diabetic Retinopathy Study (ETDRS) chart.
Time frame: 12 months
Optical coherence tomography (OCT) imaging
The number of patients with serious retinal detachment, retinal hemorrhage, and cystoid macular edema, and the change in target treatment areas.
Time frame: 12 months
Color and autofluorescence imaging
Change in target treatment areas.
Time frame: 12 months
Fluorescein angiography
Change in target treatment areas.
Time frame: 12 months
Fundus autofluorescence
Change in target treatment areas.
Time frame: 12 months
Microperimetry
Exploratory evaluations for the change of retinal sensitivity from baseline in the region of interest.
Time frame: 12 months
Electroretinography (ERG)
Exploratory evaluations for the change of retinal electrophysiology responses from baseline.
Time frame: 12 months
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