Tissue replacement in RPE related dystrophies

Phase 1

Recruiting

Conditions: Patients with retinitis pigmentosa due to monogenic mutation
MedDRA version: 20.0Level: PTClassification code: 10038914Term: Retinitis pigmentosa Class: 100000004850
Therapeutic area: Diseases [C] - Eye Diseases [C11]

Registration Number: CTIS2024-513662-18-00

Lead Sponsor: Centre D'Etude Des Cellules Souches

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 12

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess safety and tolerability of implantation of the IMP (ISTEM-01) in patients with retinitis pigmentosa over 56 weeks after IMP implantation.;Secondary Objective: To evaluate the placement and position of the patch, To assess preliminary efficacy for 2nd cohort only based on: Evaluation of visual function, Eye fundus, Evaluation of photoreceptor survival, To assess long-term safety, tolerability and efficacy (for 2nd cohort) up to 5 years after IMP implantation.;Primary end point(s): Safety and tolerability measured by the incidence of adverse events (AE) or serious adverse events (SAE) evaluated by changes in ophthalmologic exams, laboratory parameters, vital signs and in the physical examination from baseline to each visit, will be evaluated for each patient over 56 weeks.

Secondary Outcome Measures