NCT04284293

Clinical Study to Assess Safety and Efficacy of Subretinal Injection of Human Neural Progenitor Cells for Treatment of Retinitis Pigmentosa

Active, enrolled Phase 1 Last updated 14 August 2025

What this trial tests

Phase 1 trial testing CNS10-NPC implantation in Retinitis Pigmentosa in 16 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

22 July 2021

Primary endpoint
1 July 2026

1 July 2026

Quick facts

Lead sponsor	Cedars-Sinai Medical Center
Phase	Phase 1
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	16
Start date	22 July 2021
Primary completion	1 July 2026
Estimated completion	1 July 2026
Sites	1 location across United States

Drugs / interventions tested

• CNS10-NPC implantation — full drug profile →

Conditions studied

• Retinitis Pigmentosa — all drugs for Retinitis Pigmentosa →

Sponsor

Cedars-Sinai Medical Center

Who can join

18 and older, any sex, with Retinitis Pigmentosa. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Safety as evaluated by incidence of Adverse Events (AE) and Serious Adverse Events (SAE) and their relationship to the intervention
  Time frame: Subjects will be followed postoperatively for 12 months
• Safety, as evaluated by changes in the complete blood count
  Time frame: Subjects will be followed postoperatively for 12 months
• Safety, as evaluated by changes in the comprehensive metabolic panel
  Time frame: Subjects will be followed postoperatively for 12 months
• Safety, as evaluated by changes in Donor Specific Antibodies
  Time frame: Subjects will be followed postoperatively for 12 months
• Safety, as evaluated by changes in the urinalysis
  Time frame: Subjects will be followed postoperatively for 12 months
• Safety, as evaluated by changes in Visual Acuity
  Time frame: Subjects will be followed postoperatively for 12 months
  ETRDS, or FrACT in cases of very low vision.

Sponsor's own description

The investigator is examining the safety of transplanting cells into the subretinal space of patients with Retinitis Pigmentosa (RP). The cells are called neural progenitor cells, which are a type of stem cell that can become several different types of cells in the nervous system. These cells have been derived to specifically become astrocytes, which is a type of neuronal cell. The cells are called "CNS10-NPC." The investigational treatment has been tested in animals, but it has not yet been tested in people. In this study, the investigators want to learn if CNS10-NPC cells are safe to transplant into the subretinal space of people.

Publications & conference data

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

1. Advancing cell therapy for neurodegenerative diseases.
   Temple S. · · 2023 · cited 147× · PMID 37084729 · DOI 10.1016/j.stem.2023.03.017
2. Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial.
   Baloh RH, Johnson JP, Avalos P, Allred P, et al · · 2022 · cited 108× · PMID 36064599 · DOI 10.1038/s41591-022-01956-3
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. Challenges and Opportunities of Targeting Astrocytes to Halt Neurodegenerative Disorders.
   Valori CF, Possenti A, Brambilla L, Rossi D. · · 2021 · cited 26× · PMID 34440788 · DOI 10.3390/cells10082019
5. Regenerative treatment of ophthalmic diseases with stem cells: Principles, progress, and challenges.
   Niu Y, Ji J, Yao K, Fu Q. · · 2024 · cited 25× · PMID 38586868 · DOI 10.1016/j.aopr.2024.02.001
6. Human Neural Stem Cells for Cell-Based Medicinal Products.
   Fernandez-Muñoz B, Garcia-Delgado AB, Arribas-Arribas B, Sanchez-Pernaute R. · · 2021 · cited 24× · PMID 34572024 · DOI 10.3390/cells10092377
7. Pluripotent stem cell therapy for retinal diseases.
   Ahmed I, Johnston RJ, Singh MS. · · 2021 · cited 23× · PMID 34532416 · DOI 10.21037/atm-20-4747
8. Neural Cells for Neurodegenerative Diseases in Clinical Trials.
   Fan Y, Goh ELK, Chan JKY. · · 2023 · cited 17× · PMID 37487111 · DOI 10.1093/stcltm/szad041