Safety and Efficacy of Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium in the Treatment of Macular Degeneration
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Macular Degeneration
- Sponsor
- Beijing Tongren Hospital
- Enrollment
- 10
- Locations
- 1
- Primary Endpoint
- Safety measure
- Status
- Recruiting
- Last Updated
- 3 years ago
Overview
Brief Summary
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.
Detailed Description
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Aged 50-75 years;
- •Clinical diagnosis is consistent with the definition of late dry AMD in the age-related eye disease study (AREDS), with one or more \>250 um geographic atrophy in the fovea;
- •Clinical diagnosis is wet AMD, but no obvious efficacy after conventional treatment;
- •The BCVA of the target eye will be 0.05 to 0.3;
- •Voluntary as test subjects, informed consent, regular follow-up on time.
Exclusion Criteria
- •One-eyed subjects;
- •Macular atrophy caused by other diseases in addition to AMD;
- •Suffer from retinitis pigmentosa, choroidal retinitis, central serous choroiditis, diabetic retinopathy, or other retinal vascular and degenerative diseases besides AMD;
- •Lens opacities (affecting the central vision), glaucoma, uveitis, retinal detachment, optic neuropathy, and other ocular histories;
- •Other intraocular surgery histories besides cataract surgery;
- •Combined with severe systemic diseases, such as heart failure, liver disease, renal insufficiency, cor pulmonale, COPD in the previous 12 months;
- •Combined with severe infectious diseases, such as HIV, HBV, HCV, syphilis, tuberculosis, etc;
- •Abnormal blood coagulation function or other laboratory tests;
- •If female and of childbearing potential, pregnant, breastfeeding, or planning to become pregnant through the study;
- •If male, refuse to use barrier and spermicide contraception during the study;
Outcomes
Primary Outcomes
Safety measure
Time Frame: 12 months
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.
Secondary Outcomes
- Best Corrected Visual Acuity (BCVA)(12 months)
- Optical coherence tomography (OCT) imaging(12 months)
- Electroretinography (ERG)(12 months)
- Color and autofluorescence imaging(12 months)
- Fundus autofluorescence(12 months)
- Fluorescein angiography(12 months)
- Microperimetry(12 months)