NCT03963154

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

STREAM: A Phase 1/2, Open-label, Safety, Tolerability and Preliminary Efficacy Study of Implantation Into One Eye of hESC-derived RPE in Patients With Retinitis Pigmentosa Due to Monogenic Mutation

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Centre d'Etude des Cellules Souches · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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:

Detailed 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 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.

Conditions

• Retinitis Pigmentosa

Interventions

Type	Name	Description
BIOLOGICAL	Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE)	Implantation into one eye of hESC-derived RPE (Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE))

Timeline

• Start date: 2019-08-19
• Primary completion: 2023-06-22
• Completion: 2026-12-15
• First posted: 2019-05-24
• Last updated: 2023-11-28

Locations

1 site across 1 country: France