ISTEM-01 (hESC-derived retinal pigment epithelium cells) for Retinitis pigmentosa|RPE-related retinal dystrophy

Inclusion criteria

• {"criterion_text":"- 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\n- 18 years old ≤ Age ≤ 65 years old\n- During the SARS-Cov-2 pandemic period, patient negative to SARS-CoV-2 (PCR or any new test validated)\n- 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

• {"criterion_text":"- History of allergy or sensitivity to one of the products used during the study\n- Prior treatment with a gene or cell therapy product\n- 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\n- Presence of any ocular disease or ocular media opacity which in the opinion of the investigator precludes accurate evaluation\n- Patients treated with Luxturna®\n- Systemic corticosteroid therapy or other immunosuppressive/ immunomodulating or anti-retroviral drugs within 2 months prior to baseline"}

Primary endpoints

• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Measured by the incidence of adverse events (AE) or serious adverse events (SAE) evaluated by changes in ophthalmologic exams, laboratory parameters, vital signs and physical examination from baseline to each visit, evaluated for each patient over 56 weeks."}

Secondary endpoints

• {"endpoint_text":"- Placement and position of the therapeutic patch by serial spectral domain Ocular Coherence Tomography (OCT) scan at Baseline, and by study visit","definition_or_measurement_approach":"Placement and position assessed by serial spectral domain OCT at Baseline and by study visit."}
• {"endpoint_text":"- Change in leakage or perfusion in normal fundal vasculature and presence of abnormal vasculature by fundus fluorescein angiography at Baseline, weeks 24 and 56 and yearly during the long-term follow-up study.","definition_or_measurement_approach":"Assessed by fundus fluorescein angiography at Baseline, weeks 24 and 56 and annually during long-term follow-up."}
• {"endpoint_text":"- Change in thickness of RPE layer by B-mode orbital ultrasound at Baseline, weeks 24, and 56 and yearly during the long-term follow-up study.","definition_or_measurement_approach":"Measured by B-mode orbital ultrasound at Baseline, weeks 24 and 56 and annually during long-term follow-up."}
• {"endpoint_text":"- Change in eye exam and IntraOcular Pressure (IOP) from baseline, and by study visit","definition_or_measurement_approach":"Change in ocular examination findings and IOP measured from baseline and at each study visit."}
• {"endpoint_text":"- Change in ETDRS/ best corrected visual acuity (BCVA) from baseline and by study visit","definition_or_measurement_approach":"Change in ETDRS/BCVA measured from baseline and at each study visit."}
• {"endpoint_text":"- Change in kinetic perimetry from baseline, at weeks 4, 8, 12, 24, 36, 48 and 56 and yearly during the long-term follow-up study.","definition_or_measurement_approach":"Kinetic perimetry assessed at baseline, weeks 4, 8, 12, 24, 36, 48 and 56 and annually during long-term follow-up."}
• {"endpoint_text":"- Evidence of retinal and RPE functionnality by global, pattern and multifocal ERG at baseline and week 56 and yearly during the long term follow-up period","definition_or_measurement_approach":"Retinal and RPE function assessed by global, pattern and multifocal ERG at baseline, week 56 and annually during long-term follow-up."}
• {"endpoint_text":"- Functional survival of retinal photoreceptors by microperimetry over hESC-derived RPE at weeks 4, 8, 12, 24, 36, 48 and 56 and yearly during the long term follow-up period","definition_or_measurement_approach":"Microperimetry to assess functional survival of photoreceptors at weeks 4, 8, 12, 24, 36, 48 and 56 and annually during long-term follow-up."}

France

Earliest CTIS Part Ii Submission Date

24-05-2024

Latest Decision Or Authorization Date

12-06-2024

Processing Time Days

19

Number Of Sites

1

Number Of Participants

12

Primary sponsor

Full Name

Centre D'Etude Des Cellules Souches

Organisation Type

Pharmaceutical company

Country Of Registered Address

France

Third parties

• {"country":"France","full_name":"Genethon","duties_or_roles":"sponsorDuties codes: 11, 12","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"France","full_name":"ICTA Project Management En Abrege ICTA P.M.","duties_or_roles":"sponsorDuties codes: 1, 10, 11, 12, 5, 6, 7, 8","organisation_type":"Pharmaceutical company"}