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 |
|
| Source weblink |
https://clinicaltrials.gov/ct2/show/NCT03963154 |
| Public contact |
| Email |
mpeschanski@istem.fr |
| Public email |
mpeschanski@istem.fr |
| First name |
Marc |
| Last name |
Peschanski |
| Phone |
+33(0)1 69908522 |
| Country |
|
|
| Sponsors |
Centre d'Etude des Cellules Souches
|
Cells |
| Source pluripotent stem cell lines |
|
| Which differentiated cell type is used |
| Label |
dendritic cell, human |
| Link |
http://purl.obolibrary.org/obo/CL_0001056 |
| Description |
A dendritic cell with the phenotype HLA-DRA-positive.; This cell type is compatible with the HIPC Lyoplate markers for 'dendritic cell'. The inclusion of HLA-DRA in the definition restricts this definition to human dendritic cells. |
|
Recruitment |
| Recruitment Status |
Active, not recruiting |
| Comment recruitment status |
[Actual] |
| Estimated number of participants |
7 |
