Tissue replacement in RPE related dystrophies
Key facts
- Where
- Paris, FranciaországSee the site
- Phase
- 1/2. fázis
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 64 years old?
Where in Franciaország
- Quinze-Vingts National Ophthalmology HospitalParis, FranciaországTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Centre D'Etude Des Cellules Souches
- Trial code
- EU 2024-513662-18-00
- Record updated
- 2025. április 2.
- Original description
To assess safety and tolerability of implantation of the IMP (ISTEM-01) in patients with retinitis pigmentosa over 56 weeks after IMP implantation.Read full original descriptionShow less
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
To assess safety and tolerability of implantation of the IMP (ISTEM-01) in patients with retinitis pigmentosa over 56 weeks after IMP implantation.
Inclusion: 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: Prior treatment with a gene or cell therapy product; Patients treated with Luxturna®; Systemic corticosteroid therapy or other immunosuppressive/ immunomodulating or anti-retroviral drugs within 2 months prior to baseline; History of allergy or sensitivity to one of the products used during the study; 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.