RECRUITING

Safety of a Single, Intravitreal Injection of 8.8M jCell (Famzeretcel) in Retinitis Pigmentosa (RP)

Conditions

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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

This study evaluates the safety of a single injection of jCell (famzeretcel) comprising 8.8 million (8.8M) retinal progenitor cells over a six-month study period in a cohort of adult subjects with RP. Additionally, changes in visual function will be evaluated at six months between the active treatment group (8.8M jCell) compared to sham-treated controls.

Official Title

A Randomized, Masked, Sham-Controlled Phase 2 Trial of the Safety of a Single Intravitreal Injection of jCell (Famzeretcel) for the Treatment of Retinitis Pigmentosa (RP)

Quick Facts

Study Start:2025-04

Study Completion:2026-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06912633

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 to 60 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT

Inclusion Criteria	Exclusion Criteria
1. Clinical diagnosis of RP supported by at least 2 of the following clinical findings: (1) Loss of peripheral vision on formal visual field testing, (2) Symptoms of night blindness or difficulty adjusting to dim light, or (3) Optical coherence tomography (OCT) outer retinal atrophy consistent with RP. 2. Electroretinography (ERG) results that support diagnosis of RP including nondetectable or severely reduced rod responses (defined as less than 30% of the lower limit of normative values for the ERG lab performing the test), with prolonged implicit time OU (i.e., ensure bilateral involvement) and greater rod than cone loss. 3. Subject age ≥ 18 years and ≤ 60 years at time of signing of consent. 4. Interocular BCVA disparity ≤ 15 letters. 5. Central subfield thickness (CST) ≥ 130 µm in the study eye. 6. BCVA no better than 55 letters and no worse than 1 letter using the Early Treatment Diabetic Retinopathy Study (ETDRS) testing protocol in the study eye. 7. Ability to reliably fixate with the study eye at least 75% of the time as indicated by a fixation score of four (4) or five (5) on semi-automated kinetic visual fields. 8. Ability to record at least two reliable trials at a minimum baseline contrast sensitivity reading of 1.28 at a minimum of one spatial frequency using the Beethoven system in the study eye. 9. Central island visual field area (central island contiguous to fixation), of ≥ 50.3 deg2 (\~ central island visual field diameter ≥ 8°) in the study eye as assessed by semi-automated kinetic visual field (KVF) using the Octopus 900 (target size V4e), with the reaction times turned off. 10. Willingness of subject to provide informed consent, including acknowledgement that they are able and willing to attend all required study visits, follow study protocol assessment instructions, travel by air if necessary, and provide Health Insurance Portability and Accountability Act (HIPAA) authorization. 11. Willingness of subject to provide a blood sample for human leukocyte antigen (HLA) typing, if not done previously with available results. 12. Willingness of subject to consent to testing for RP gene mutation typing, if not performed previously with available results. 13. Adequate organ function. 14. Negative active infectious disease screen (active infection with Hepatitis B, C, human immunodeficiency virus \[HIV\]). 15. A female subject of childbearing potential must have a negative pregnancy test (urine human chorionic gonadotropin) at entry (prior to treatment). 16. Women of childbearing potential must agree to use a medically accepted method of contraception for at least 12 months after jCell injection. 17. For male patients whose partners are of child-bearing potential, willingness to use a medically accepted method of contraception.	1. Participation in any clinical trial of a drug intervention within the last 6 months, with the exception of a N-acetyl cysteine (NAC) study. 2. History of ocular treatment with any non-approved, experimental, investigational or neuroprotectant therapy (systemic, topical, intravitreal) or device in either eye, including previous jCyte clinical trials. Individuals with a history of NAC treatment may take part in the study following a 7-day washout period (prior to Baseline testing). 3. Subject is currently breast feeding/pumping or is planning to breast feed/pump during the 12 months after study treatment. 4. Subject is pregnant or intends to become pregnant less than 12 months after jCell injection. 5. Known allergy to gentamicin. 6. History of adverse reaction to dimethyl sulfoxide (DMSO). 7. Prior ocular treatment with corticosteroids (systemic, periocular, intracanalicular or intravitreal - in either eye) within six months of study randomization or the anticipated need for the use of these agents to treat a pre-existing ocular condition. 8. Clinical evidence of history of any eye disease or pathology, other than RP, IN EITHER EYE, that is associated with increased risk of pathology developing in the study eye, that could impair visual function, testing procedures, clinical trial endpoint measurements and/or the outcome of the study. 9. Clinical evidence of history of any eye disease or pathology, other than RP, IN THE STUDY EYE, that could potentially impair visual function, testing procedures, clinical trial endpoint measurements and/or the outcome of the study. 10. Concurrent use of any prohibited therapies. 11. History of prior use of the following medications is prohibited if any retinal/retinal pigment epithelium (RPE) abnormalities are noted in the macula on exam or OCT: Hydroxychloroquine or chloroquine (Plaquenil); Pentosan polysulfate sodium \[PPS\] (Elmiron); and Interferon (Intron A, Roferon-A, IFN-alpha, alpha interferon). 12. Any mental health issue likely to prevent subject from reliably performing study testing and/or examinations including dementia, schizophrenia, bipolar disorders if not reliably controlled on medications, depression if any history of hospitalization or in-patient treatment or if not sufficiently controlled on medications to enable, in the opinion of the investigator, travel to and compliance with study testing requirements over the study period. 13. Uncontrolled blood pressure defined as systolic pressure \> 180mmHg and/or diastolic blood pressure \> 110mmHg, while subject is at rest. 14. Any chronic systemic disease requiring continuous treatment with systemic steroids or immunosuppressive agents. 15. History of any disease interfering with the participation in the study according to the investigator judgment, including of any type of cancer that is not in remission or considered cured, diabetes mellitus (history of gestational diabetes is acceptable), renal failure, stroke, transient ischemic attack (TIA), any systemic immune condition, any coagulopathy disorder that is not adequately managed/controlled. 16. Current systemic treatment for a confirmed active infection. 17. For male patients whose co-partners are of child-bearing potential, lack of willingness to use a medically accepted method of contraception, not including the rhythm method, for at least 12 months after jCell injection.

Inclusion Criteria

Exclusion Criteria

1. Clinical diagnosis of RP supported by at least 2 of the following clinical findings: (1) Loss of peripheral vision on formal visual field testing, (2) Symptoms of night blindness or difficulty adjusting to dim light, or (3) Optical coherence tomography (OCT) outer retinal atrophy consistent with RP.
2. Electroretinography (ERG) results that support diagnosis of RP including nondetectable or severely reduced rod responses (defined as less than 30% of the lower limit of normative values for the ERG lab performing the test), with prolonged implicit time OU (i.e., ensure bilateral involvement) and greater rod than cone loss.
3. Subject age ≥ 18 years and ≤ 60 years at time of signing of consent.
4. Interocular BCVA disparity ≤ 15 letters.
5. Central subfield thickness (CST) ≥ 130 µm in the study eye.
6. BCVA no better than 55 letters and no worse than 1 letter using the Early Treatment Diabetic Retinopathy Study (ETDRS) testing protocol in the study eye.
7. Ability to reliably fixate with the study eye at least 75% of the time as indicated by a fixation score of four (4) or five (5) on semi-automated kinetic visual fields.
8. Ability to record at least two reliable trials at a minimum baseline contrast sensitivity reading of 1.28 at a minimum of one spatial frequency using the Beethoven system in the study eye.
9. Central island visual field area (central island contiguous to fixation), of ≥ 50.3 deg2 (\~ central island visual field diameter ≥ 8°) in the study eye as assessed by semi-automated kinetic visual field (KVF) using the Octopus 900 (target size V4e), with the reaction times turned off.
10. Willingness of subject to provide informed consent, including acknowledgement that they are able and willing to attend all required study visits, follow study protocol assessment instructions, travel by air if necessary, and provide Health Insurance Portability and Accountability Act (HIPAA) authorization.
11. Willingness of subject to provide a blood sample for human leukocyte antigen (HLA) typing, if not done previously with available results.
12. Willingness of subject to consent to testing for RP gene mutation typing, if not performed previously with available results.
13. Adequate organ function.
14. Negative active infectious disease screen (active infection with Hepatitis B, C, human immunodeficiency virus \[HIV\]).
15. A female subject of childbearing potential must have a negative pregnancy test (urine human chorionic gonadotropin) at entry (prior to treatment).
16. Women of childbearing potential must agree to use a medically accepted method of contraception for at least 12 months after jCell injection.
17. For male patients whose partners are of child-bearing potential, willingness to use a medically accepted method of contraception.

1. Participation in any clinical trial of a drug intervention within the last 6 months, with the exception of a N-acetyl cysteine (NAC) study.
2. History of ocular treatment with any non-approved, experimental, investigational or neuroprotectant therapy (systemic, topical, intravitreal) or device in either eye, including previous jCyte clinical trials. Individuals with a history of NAC treatment may take part in the study following a 7-day washout period (prior to Baseline testing).
3. Subject is currently breast feeding/pumping or is planning to breast feed/pump during the 12 months after study treatment.
4. Subject is pregnant or intends to become pregnant less than 12 months after jCell injection.
5. Known allergy to gentamicin.
6. History of adverse reaction to dimethyl sulfoxide (DMSO).
7. Prior ocular treatment with corticosteroids (systemic, periocular, intracanalicular or intravitreal - in either eye) within six months of study randomization or the anticipated need for the use of these agents to treat a pre-existing ocular condition.
8. Clinical evidence of history of any eye disease or pathology, other than RP, IN EITHER EYE, that is associated with increased risk of pathology developing in the study eye, that could impair visual function, testing procedures, clinical trial endpoint measurements and/or the outcome of the study.
9. Clinical evidence of history of any eye disease or pathology, other than RP, IN THE STUDY EYE, that could potentially impair visual function, testing procedures, clinical trial endpoint measurements and/or the outcome of the study.
10. Concurrent use of any prohibited therapies.
11. History of prior use of the following medications is prohibited if any retinal/retinal pigment epithelium (RPE) abnormalities are noted in the macula on exam or OCT: Hydroxychloroquine or chloroquine (Plaquenil); Pentosan polysulfate sodium \[PPS\] (Elmiron); and Interferon (Intron A, Roferon-A, IFN-alpha, alpha interferon).
12. Any mental health issue likely to prevent subject from reliably performing study testing and/or examinations including dementia, schizophrenia, bipolar disorders if not reliably controlled on medications, depression if any history of hospitalization or in-patient treatment or if not sufficiently controlled on medications to enable, in the opinion of the investigator, travel to and compliance with study testing requirements over the study period.
13. Uncontrolled blood pressure defined as systolic pressure \> 180mmHg and/or diastolic blood pressure \> 110mmHg, while subject is at rest.
14. Any chronic systemic disease requiring continuous treatment with systemic steroids or immunosuppressive agents.
15. History of any disease interfering with the participation in the study according to the investigator judgment, including of any type of cancer that is not in remission or considered cured, diabetes mellitus (history of gestational diabetes is acceptable), renal failure, stroke, transient ischemic attack (TIA), any systemic immune condition, any coagulopathy disorder that is not adequately managed/controlled.
16. Current systemic treatment for a confirmed active infection.
17. For male patients whose co-partners are of child-bearing potential, lack of willingness to use a medically accepted method of contraception, not including the rhythm method, for at least 12 months after jCell injection.

Contacts and Locations

Study Contact

jCyte Sr. Director of Clinical Operations

CONTACT

949-688-1816

info@jcyte.com

Principal Investigator

Henry Klassen, MD, PhD

PRINCIPAL_INVESTIGATOR

jCyte, Inc

Study Locations (Sites)

Retina-Vitreous Associates Medical Group

Beverly Hills, California, 90074

United States

Gavin Herbert Eye Institute, UC Irvine

Irvine, California, 92697

United States

Collaborators and Investigators

Sponsor: jCyte, Inc

Henry Klassen, MD, PhD, PRINCIPAL_INVESTIGATOR, jCyte, Inc

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 Date2025-04

Study Completion Date2026-01

Study Record Updates

Study Start Date2025-04

Study Completion Date2026-01

Terms related to this study

Additional Relevant MeSH Terms

Retinitis Pigmentosa