A phase 1/2, open-label, safety, tolerability and preliminary efficacy study of implantation into one eye of hESC-derived RPE (Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE)) in patients with retinitis pigmentosa due to monogenic mutation - STREAM

Centre D'Etude Des Cellules Souches0 个研究点目标入组 12 人开始时间: 2024年5月15日最近更新: 2024年8月21日

适应症

试验速览

暂无简介。

•Documented diagnosis of retinis pigmentosa based on a genetic test confirming the presence of a monogenic mutation that affects a gene involved in the visual signalling process specifically at the level of RPEs, namely RPE65 or LRAT, or MerTK, 18 years old = Age = 65 years old, During the SARS-Cov-2 pandemic period, patient negative to SARS-CoV-2 (PCR or any new test validated), For patient of the first cohort: Visual acuity = 20/200 in the best eye (legally blind). For patient of the second cohort: 20/63 > Visual acuity > 20/800 in the worst eye And Visual field exceeding 10° central to the V4 test in the worst eye And Visible photoreceptor outer nuclear layer (ONL) on a spectral domain optical coherence tomography (OCT) scan

•History of allergy or sensitivity to one of the products used during the study, Prior treatment with a gene or cell therapy product, Patients with preformed anti-HLA antibodies against the human Embryonic Stem Cell (hESC) line RC9 with MFI > 2000 or patients with intermediate MFI values (between 500 and 2000) at screening associated to medical history of clinical significance, according to expert’s opinion, Presence of any ocular disease or ocular media opacity which in the opinion of the investigator precludes accurate evaluation, Patients treated with Luxturna®, Systemic corticosteroid therapy or other immunosuppressive/ immunomodulating or anti-retroviral drugs within 2 months prior to baseline

发起方

Centre D'Etude Des Cellules Souches