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

NCT ID: NCT06912633

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-06-20

Study Completion Date

2026-09-30

Brief Summary

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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.

Detailed Description

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Conditions

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Retinitis Pigmentosa

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Outcome Assessors
Subjects, their family members, and clinical staff performing efficacy assessments will be masked to the randomization assignment of subjects. Due to the nature of some safety assessments and the sham treatment, not all study personnel can be masked.

Study Groups

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8.8M jCell injection

Single intravitreal injection of 8.8 million retinal progenitor cells into the study eye

Group Type EXPERIMENTAL

human retinal progenitor cells

Intervention Type BIOLOGICAL

Single intravitreal injection of 8.8 million retinal progenitor cells (RPCs)

Sham-treated control

A mock injection will be performed on the study eye in each control subject

Group Type SHAM_COMPARATOR

Mock injection

Intervention Type OTHER

Pressing the hub of a syringe with no needle against the eye to mimic intravitreal injection. Subjects randomized to the sham control group will undergo identical preparation as the active treatment group immediately prior to treatment, including application of anesthetics.

Interventions

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human retinal progenitor cells

Single intravitreal injection of 8.8 million retinal progenitor cells (RPCs)

Intervention Type BIOLOGICAL

Mock injection

Pressing the hub of a syringe with no needle against the eye to mimic intravitreal injection. Subjects randomized to the sham control group will undergo identical preparation as the active treatment group immediately prior to treatment, including application of anesthetics.

Intervention Type OTHER

Other Intervention Names

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jCell famzeretcel

Eligibility Criteria

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Inclusion 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.

Exclusion Criteria

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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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jCyte, Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Henry Klassen, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

jCyte, Inc

Locations

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California Retina Consultants

Bakersfield, California, United States

Site Status RECRUITING

Retina-Vitreous Associates Medical Group

Beverly Hills, California, United States

Site Status RECRUITING

Gavin Herbert Eye Institute, UC Irvine

Irvine, California, United States

Site Status RECRUITING

Retina Consultants Medical Group

Sacramento, California, United States

Site Status RECRUITING

Shiley Eye Institute, UC San Diego

San Diego, California, United States

Site Status RECRUITING

Illinois Retina Associates

Oak Park, Illinois, United States

Site Status RECRUITING

Long Island Vitreoretinal Consultants

Westbury, New York, United States

Site Status RECRUITING

Retina Consultants of Texas: Bellaire Retina Center

Bellaire, Texas, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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jCyte Sr. Director of Clinical Operations

Role: CONTACT

Phone: 949-688-1816

Email: [email protected]

Facility Contacts

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Study Coordinator

Role: primary

Clinical Trials Coordinator

Role: primary

Study Coordinator

Role: primary

Study Coordinator

Role: primary

Study Coordinator

Role: primary

Role: backup

RCA Research Referrals Center

Role: primary

Ewelina Lokaj

Role: primary

Study Coordinator

Role: primary

Other Identifiers

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JC02-88

Identifier Type: -

Identifier Source: org_study_id