Phase I/II 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. Study non randomized single group assignment consisting in 2 sequential cohorts of patients: * First cohort of 2 patients with very advanced loss of visual acuity (legally blind) * Second cohort of 10 patients with less advanced loss of visual acuity:
Monocentric study (Hospital of 15-20 at Paris) of duration of 106 weeks. At total,12 evaluable patients will be enrolled and assigned in 2 cohorts as described above in brief summary. Expected follow-up for one patient is about 64 weeks including 8 weeks of screening and baseline period and 56 weeks of follow-up after implantation of hESC-derived RPE. After 56 weeks of follow-up, patients will be enrolled in a long term follow-up study during 4 additional years. The primary objective is to assess safety and tolerability of implantation of the Investigational Medecinal Product (ISTEM-01) in patients with retinitis pigmentosa. Secondary objectives are: * To evaluate the placement and position of the patch * To assess preliminary efficacy based on: * Evaluation of visual function * Eye fundus * Evaluation of photoreceptor survival Assessment of visual function by Diagnosys-Full-field stimulus threshold (D-FST) is the only exploratory objective.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
7
Implantation into one eye of hESC-derived RPE (Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE))
•Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts
Paris, France
Safety assessment
Safety and tolerability measured by the incidence of Adverse Events or Serious Adverse Events throughout the study
Time frame: From Baseline until Week 56
Position of therapeutic patch
Position of the therapeutic patch by serial images assessment
Time frame: From baseline until Week 56
Placement of the therapeutic patch
Placement of the therapeutic patch by serial spectral domain Ocular Coherence Tomography (OCT) scan at baseline and by study visit
Time frame: From Baseline until Week 56
Change in leakage or perfusion
Change in leakage or perfusion in normal fundal vasculature and presence of abnormal vasculature by fundus fluorescein angiography
Time frame: At baseline and weeks 24, 48, and 56
Change in thickness of RPE layer
Change in thickness of RPE layer by B-mode orbital ultrasound
Time frame: At weeks 4, 8, 16, 24, 36, 48 and 56
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