drugset / Trial / NCT03963154

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

NCT03963154 ↗

Phase 1/2 Active not recruiting 7 enrolled Centre d'Etude des Cellules Souches
NaSequentialOpen-labelTreatment

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 acuity:

Timeline

Start
2019-08-19
Primary completion
2023-06-22
Completion
2026-12-15

Drugs

EvaluationDrugModalityDoseRoute
Subject ISTEM-01 Cell therapy — Other

Indications