NCT03963154

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

Active, enrolled Phase 1, PHASE2 Last updated 28 November 2023

What this trial tests

Phase 1, PHASE2 trial testing Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE) in Retinitis Pigmentosa in 7 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

19 August 2019

Primary endpoint
22 June 2023

15 December 2026

Quick facts

Lead sponsor	Centre d'Etude des Cellules Souches
Phase	Phase 1, PHASE2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	7
Start date	19 August 2019
Primary completion	22 June 2023
Estimated completion	15 December 2026
Sites	1 location across France

Drugs / interventions tested

• Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE) — full drug profile →

Conditions studied

• Retinitis Pigmentosa — all drugs for Retinitis Pigmentosa →

Sponsor

Centre d'Etude des Cellules Souches — full company profile →

Who can join

Adults 18 to 65, any sex, with Retinitis Pigmentosa. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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:

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Global trends in clinical trials involving pluripotent stem cells: a systematic multi-database analysis.
   Deinsberger J, Reisinger D, Weber B. · · 2020 · cited 109× · PMID 32983575 · DOI 10.1038/s41536-020-00100-4
2. Immunological considerations and challenges for regenerative cellular therapies.
   Petrus-Reurer S, Romano M, Howlett S, Jones JL, et al · · 2021 · cited 97× · PMID 34172826 · DOI 10.1038/s42003-021-02237-4
3. Regenerative and restorative medicine for eye disease.
   Van Gelder RN, Chiang MF, Dyer MA, Greenwell TN, et al · · 2022 · cited 77× · PMID 35715505 · DOI 10.1038/s41591-022-01862-8
4. Stem-cell-based therapies for retinal degenerative diseases: Current challenges in the establishment of new treatment strategies.
   Uyama H, Mandai M, Takahashi M. · · 2021 · cited 37× · PMID 33315237 · DOI 10.1111/dgd.12704
5. Advancements in Human Embryonic Stem Cell Research: Clinical Applications and Ethical Issues.
   Park SJ, Kim YY, Han JY, Kim SW, et al · · 2024 · cited 29× · PMID 38502279 · DOI 10.1007/s13770-024-00627-3
6. Stem cell therapy in retinal diseases.
   Voisin A, Pénaguin A, Gaillard A, Leveziel N. · · 2023 · cited 28× · PMID 36571345 · DOI 10.4103/1673-5374.361537
7. Recent Progress in Retinal Pigment Epithelium Cell-Based Therapy for Retinal Disease.
   Klymenko V, González Martínez OG, Zarbin M. · · 2024 · cited 27× · PMID 38394392 · DOI 10.1093/stcltm/szae004
8. Cell Replacement Therapy for Retinal and Optic Nerve Diseases: Cell Sources, Clinical Trials and Challenges.
   Coco-Martin RM, Pastor-Idoate S, Pastor JC. · · 2021 · cited 26× · PMID 34208272 · DOI 10.3390/pharmaceutics13060865