General Information |
| 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 acuit. |
| 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.
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. |
| Clinical trials phase |
Phases 1/2 |
| Start date (estimated) |
2019-08-19 |
| End date (estimated) |
2026-12-15 |
| Clinical feature |
| Label |
retinitis pigmentosa |
| Link |
http://purl.obolibrary.org/obo/DOID_10584 |
| Description |
A retinal degeneration characterized by the gradual deterioration of the photoreceptors or the retinal pigment epithelium of the retina leading to progressive sight loss.; Xref MGI. OMIM mapping confirmed by DO. [LS]. |
|
Administrative Information |
| NCT number |
NCT03963154 |
| ICTRP weblink |
https://trialsearch.who.int/Trial2.aspx?TrialID=NCT03963154 |
| Other study identifiers |
| Name |
CTIS2024-513662-18-00 |
| Description |
Clinical Trials Information System (CTIS) |
|
| Source weblink |
https://clinicaltrials.gov/ct2/show/NCT03963154 |
| Sponsors |
Centre d'Etude des Cellules Souches
|
Cells |
| Source pluripotent stem cell lines |
|
| Which differentiated cell type is used |
| Label |
retinal pigment epithelial cell |
| Link |
http://purl.obolibrary.org/obo/CL_0002586 |
| Description |
An epithelial cell of the retinal pigmented epithelium.; Retinal pigment epithelial (RPE) cells form a single layer of cells at the back of the eye sandwiched between the neurosensory retina and the choroid, playing a significant role in maintaining vision health. These pigment-laden cells are highly specialized and perform an array of metabolic and transport functions essential for the maintenance of the photoreceptor cells (rods and cones) in the retina. The pigmentation of RPE cells actively aids in the absorption of excess light and the prevention of light scattering, thus enhancing the eye's optical properties.
The retinal pigment epithelium forms a key part of the blood/retina barrier. The cells have long sheet-like microvilli on their apical membrane that project into the light-sensitive outer segments of the photoreceptors, forming a close structural interaction. The basolateral membrane of the RPE interacts with the underlying Bruch’s membrane, which separates the RPE cells from fenestrated endothelium of the choriocapillaris.
RPE cells support the photoreceptor by providing them with oxygen and nutrients (such as glucose, retinol and fatty acids) and removing waste products. They also recycle the visual pigment, in a process called the "visual cycle", where the RPE cells play a vital role in the regeneration of visual pigment (11-cis retinol) following the absorption of light. This is essential for the maintenance of photoreceptor excitability.
Beyond this, RPE cells take part in the phagocytosis process, where they digest the shed ends of photoreceptor outer segments, thus, preventing the build-up of waste residue that could otherwise harm retinal health. They also secrete various factors, including growth factors required to maintain the structural integrity of choriocapillaris endothelium and photoreceptors, as well as immunosuppressive factors that play an important role in establishing the immune privilege of the eye.
(This extended description was generated by ChatGPT and reviewed by the CellGuide team, who added references, and by the CL editors, who approved it for inclusion in CL. It may contain information that applies only to some subtypes and species, and so should not be considered definitional.) |
|
Recruitment |
| Recruitment Status |
Active, not recruiting |
| Comment recruitment status |
[Actual] |
| Estimated number of participants |
7 |
