Transplanting neural progenitor cells to treat Retinitis Pigmentosa
Official title: Clinical Study to Assess Safety and Efficacy of Subretinal Injection of Human Neural Progenitor Cells for Treatment of Retinitis Pigmentosa
Run by Cedars-Sinai Medical Center · NCT04284293 · Data updated 2026-05-14
This study is no longer in our active list. It may have stopped recruiting or finished. The details below are as of 2026-05-14 and are kept for reference. Check ClinicalTrials.gov for its current status, or see similar studies below.
This study is testing if transplanting special cells into the eyes of people with Retinitis Pigmentosa can be safe and help slow down vision loss.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 16 (estimated) |
| Sponsor | Cedars-Sinai Medical Center Academic / other |
| Locations | 1 site (Beverly Hills, California) |
| Trial ID | NCT04284293 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are adults aged 18 and older diagnosed with Retinitis Pigmentosa and experiencing significant vision impairment.
✕Probably not a fit
Patients with significant ocular abnormalities that would prevent them from undergoing the planned surgery may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This clinical trial investigates the safety of transplanting neural progenitor cells (CNS10-NPC) into the subretinal space of patients with Retinitis Pigmentosa (RP). It is a Phase 1/2a, single-center, open-label study that will assess two escalating doses of these cells delivered unilaterally. The study aims to determine the safety and tolerability of the treatment, as well as its potential to engraft and impact vision loss progression. Participants will be closely monitored for safety and any adverse effects following the cell implantation.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option to slow or halt vision loss in patients with Retinitis Pigmentosa.
How similar studies have gone: While this approach is novel and has not been tested in humans before, previous studies with similar cell therapies in animal models have shown promise.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria:
1. To participate in this study, the subject must be 18 years of age or older and must understand and sign the protocol's informed consent.
2. Participant with diagnosis of retinitis pigmentosa.
* Clinical signs of retinitis pigmentosa.
* A history of nyctalopia
* Retinal pigmentary changes
* Arteriolar attenuation
* Waxy disc pallor
* Electrophysiologic evidence of rod dysfunction on full field electroretinography
* Visual field constriction.
3a. Participants in Group 1 (n=6) will have visual acuity equal to or worse than 20/200. Participants in Group 2 (n=10) will have visual acuity equal to or worse than 20/80.
3b. Group 1 participants will have central visual field of 40 degrees diameter or less. Group 2, participants will have a measurable visual field defect.
4. Participant will be medically able to undergo ophthalmic surgery.
Exclusion Criteria:
1. Presence of significant ocular abnormalities that would preclude the planned surgery or interfere with the interpretation of study endpoints (e.g. glaucoma, corneal or significant lens opacities, pre-existing uveitis, intraocular infection, macular edema, choroidal neovascularization). Any ocular diseases that the investigator feels would interfere with accurate ocular measurements. This would exclude potential subjects with significant cataract, corneal scars or significant corneal irregularities such as keratoconus, previous penetrating keratoplasty or glaucoma with central visual field.
2. Any pre-existing factor or history of eye disease that may predispose to an increased risk of surgical complications in the study eye (e.g. trauma, previous surgery other than uncomplicated cataract surgery, uveitis, congenital developmental or structural abnormalities).
3. Concomitant systemic diseases including those in which the disease itself, or the treatment for the disease, can alter ocular function or immune status (e.g. malignancies, uncontrolled diabetes).
4. Any ocular surgery or laser in either eye within 12 weeks of screening.
5. Any contraindication to pupil dilatation in either eye.
6. Treatment with intravitreal, subtenon or periocular steroid within 4 months of enrollment.
7. Any known allergy to any component of the delivery vehicle or diagnostic agents used during the study (e.g., dilation drops), or medications planned for use during the peri-operative period including corticosteroids, tacrolimus and mycophenolate.
8. Imminently life-threatening illness.
9. Abuse of alcohol or any illegal substance(s) within 12 months of the procedure.
10. Laboratory test abnormalities or abnormalities in electrocardiogram or chest X-ray, which in the opinion of the Principal Investigator are clinically significant and would make the patient unable to tolerate study procedures.
11. Intercurrent illness or infection 28 days prior to enrolment.
12. Contraindications to use of anesthesia.
13. Females of child-bearing potential (i.e. those who are not surgically sterile and not at least 12-months post-menopausal) who are not willing to comply with the study's contraception requirement (women who are unwilling to use an effective form of contraception such as the contraceptive pill or intrauterine device).
14. Women who are pregnant.
15. Females who are nursing or who intend to nurse during the first 6 months post-treatment.
16. Men or women who do not agree to use barrier or medical contraception for at least 6 months post-operatively.
17. History of any investigational agent administration within 28 days prior to administration.
18. Participation in a prior gene transfer therapy study or cell-based therapy.
19. Enrollment in any other clinical study, for any condition, including those relating to RP throughout the duration of the study.
20. Current or anticipated long-term treatment with systemic corticosteroids (for a period longer than 7 days).
21. Current treatment with immunosuppressant therapies or any contraindications to use of the immunosuppressants in this protocol.
22. A history of malignancy within a five-year period or a positive cancer screening test within a one-year period of the screening visit.
23. Any physical or mental disability that will impair the ability of the patient to travel to and from the study center or provide informed consent/assent or effective safety assessments as specified by the protocol. Any mental or psychiatric disorders that prevent the patient from having full authority to do so (i.e. the subject cannot have power of attorney signed over to another individual).
24. Inability or unwillingness to comply with the study protocol.
25. Medical history of HIV, or hepatitis A, B or C.
26. Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane® Soriatane®), or any medicines that may affect the macula (e.g. tamoxifen, mellaril, thorazine, plaquenil, niacin) within 6 months of enrollment.
27. Allergy to Beta-Lactam antibiotics.
28. The presence of cystoid macular edema.
29. Glucose-6-phosphate dehydrogenase (G6PD) Deficiency
Where it's running
-
Retina-Vitreous Associates Medical Group
Beverly Hills, California, United States Recruiting
Study contacts
- Principal investigator: David Liao, MD, PhD — Retina-Vitreous Associates Medical Group
- Study coordinator: Pablo Avalos, MD
- Email: Pablo.Avalos@cshs.org
- Phone: 310-248-8584
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
Functional visual field and navigation testing for moderate-to-profound inherited retinal disease
This project will test whether functional visual field and navigation tests can measure real-world vision and mobility in people with moderate-to-profound inherited retinal diseases.
-
OCT-980 oral treatment for rhodopsin-associated autosomal dominant retinitis pigmentosa
This program will test whether the oral drug OCT-980 is safe, well tolerated, and shows signs of benefit in adults with RHO-associated autosomal dominant retinitis pigmentosa and in healthy volunteers.
-
Course of EYS-associated retinitis pigmentosa in adults from Russia and former CIS
We will follow adults with pathogenic EYS gene variants from Russia and former CIS for four years to try to find reliable vision and retinal measures for future treatments.
-
ZVS203e eye gene-editing therapy for a specific RHO-related retinitis pigmentosa
This trial tests a single subretinal injection of ZVS203e to see if it’s safe and can help a specific RP caused by an RHO mutation.
-
Testing IGT001 for treating Retinitis Pigmentosa
This study is testing a new treatment called IGT001 for people with retinitis pigmentosa to see if it can help improve their vision and how safe it is to use.
-
Safety of a one-time DSP-3077 retinal sheet injection in adults with retinitis pigmentosa
This will test whether a single subretinal injection of DSP-3077 retinal sheets is safe and well tolerated in adults with retinitis pigmentosa.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.