Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation

General Information

Summary 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 acuit.
Description 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. 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.
Clinical trials phase Phases 1/2
Start date (estimated) 2019-08-19
End date (estimated) 2026-12-15
Clinical feature
Label retinitis pigmentosa
Description A retinal degeneration characterized by the gradual deterioration of the photoreceptors or the retinal pigment epithelium of the retina leading to progressive sight loss.; Xref MGI. OMIM mapping confirmed by DO. [LS].

Administrative Information

NCT number NCT03963154
ICTRP weblink
Other study identifiers
Source weblink
Public contact
Public email
First name Marc
Last name Peschanski
Phone +33(0)1 69908522
Sponsors Centre d'Etude des Cellules Souches


Source pluripotent stem cell lines
Which differentiated cell type is used
Label dendritic cell, human
Description A dendritic cell with the phenotype HLA-DRA-positive.; This cell type is compatible with the HIPC Lyoplate markers for 'dendritic cell'. The inclusion of HLA-DRA in the definition restricts this definition to human dendritic cells.


Recruitment Status Active, not recruiting
Comment recruitment status [Actual]
Estimated number of participants 7