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

Retinitis Pigmentosa Clinical Trial

Official title: 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

Clinical Trial 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:

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa

• NCT number: NCT03963154
• Study type: Interventional
• Source: Centre d'Etude des Cellules Souches
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: August 19, 2019
• Completion date: December 15, 2026