ACTIVE_NOT_RECRUITING

CNS10-NPC for the Treatment of RP

Conditions

Retinitis Pigmentosa RP Retinopathy Pigmentary Retinopathy Retinal Degeneration

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2021-07-22

Study Completion:2026-07

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT04284293

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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. 4. Participant will be medically able to undergo ophthalmic surgery.	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

Inclusion Criteria

Exclusion 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.
4. Participant will be medically able to undergo ophthalmic surgery.

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

Contacts and Locations

Principal Investigator

David Liao, MD, PhD

PRINCIPAL_INVESTIGATOR

Retina-Vitreous Associates Medical Group

Study Locations (Sites)

Retina-Vitreous Associates Medical Group

Beverly Hills, California, 90211

United States

Collaborators and Investigators

Sponsor: Cedars-Sinai Medical Center

David Liao, MD, PhD, PRINCIPAL_INVESTIGATOR, Retina-Vitreous Associates Medical Group

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-07-22

Study Completion Date2026-07

Study Record Updates

Study Start Date2021-07-22

Study Completion Date2026-07

Terms related to this study

Keywords Provided by Researchers

RP
Retinopathy
Pigmentary Retinopathy
Retinal Degeneration

Additional Relevant MeSH Terms

Retinitis Pigmentosa