Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation
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|ClinicalTrials.gov Identifier: NCT03963154|
Recruitment Status : Recruiting
First Posted : May 24, 2019
Last Update Posted : June 3, 2021
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:
|Condition or disease||Intervention/treatment||Phase|
|Retinitis Pigmentosa||Biological: Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE)||Phase 1 Phase 2|
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 (Clinical Trial)|
|Estimated Enrollment :||12 participants|
|Intervention Model:||Sequential Assignment|
|Intervention Model Description:||Open-label study|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||August 19, 2019|
|Estimated Primary Completion Date :||December 15, 2022|
|Estimated Study Completion Date :||December 15, 2026|
Experimental: Implantation of a therapeutical patch
All the patients will receive a single central subretinal implantation in one eye of a monolayer of Human Embryonic Stem Cells-derived Retinal Pigmented Epithelium (hESC-derived RPE). The implanted eye will be the one with the worst visual acuity.
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))
- Safety assessment [ Time Frame: From Baseline until Week 56 ]Safety and tolerability measured by the incidence of Adverse Events or Serious Adverse Events throughout the study
- Position of therapeutic patch [ Time Frame: From baseline until Week 56 ]Position of the therapeutic patch by serial images assessment
- Placement of the therapeutic patch [ Time Frame: From Baseline until Week 56 ]Placement of the therapeutic patch by serial spectral domain Ocular Coherence Tomography (OCT) scan at baseline and by study visit
- Change in leakage or perfusion [ Time Frame: At baseline and weeks 24, 48, and 56 ]Change in leakage or perfusion in normal fundal vasculature and presence of abnormal vasculature by fundus fluorescein angiography
- Change in thickness of RPE layer [ Time Frame: At weeks 4, 8, 16, 24, 36, 48 and 56 ]Change in thickness of RPE layer by B-mode orbital ultrasound
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03963154
|Contact: Marc PESCHANSKI, MD-PhD||+33(0)1 firstname.lastname@example.org|
|Contact: Christelle MONVILLE, PhD||+33(0)1 email@example.com|
|•Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts||Recruiting|
|Paris, France, 75012|
|Contact: Stéphane BERTIN, MD|
|Principal Investigator:||Stéphane BERTIN, MD||Centre des 15-20|