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Safety and Efficacy of Autologous Transplantation of iPSC-RPE in the Treatment of Macular Degeneration

Recruiting
50 - 75 years of age
Both
Phase 1

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Overview

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.

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.

Eligibility

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;
  • Malignant tumor and history of malignancy;
  • Any immune deficiency;
  • Allergy to tacrolimus or other macrolides;
  • Any immune deficiency;
  • Use glucocorticoids, immunosuppressive drugs, or antipsychotic drugs in the previous 3 months;
  • Use anticoagulant, or the platelet function is still not restored to normal after stopping antiplatelet drugs for 10 days;
  • A history of addiction to alcoholism or prohibited drugs;
  • Be participating in other intervention clinical trials or receiving other study medications;
  • Poor compliance, difficulty to complete the study, or refusal to informed consent;
  • Some other situations which might increase the risks of the subjects or interfere with clinical trials, such as mental disorders, cognitive dysfunction, etc.

Study details

Macular Degeneration

NCT05445063

Beijing Tongren Hospital

25 January 2024

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