Safety of a Single, Intravitreal Injection of 6.0M jCell (Famzeretcel) in Retinitis Pigmentosa (RP)

NCT06912633 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: JC02-88
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 14

Brief Summary

This study evaluates the safety of a single injection of jCell (famzeretcel) comprising 6.0 million (6.0M) 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 (6.0M jCell) compared to sham-treated controls.

Conditions

Retinitis Pigmentosa

Outcome Measures

Primary Outcomes (1)

• Safety of Intravitreal Injection of 6.0M jCell

  Assessed by treatment emergent adverse events, immunogenicity and safety visual assessments

  6 months

Secondary Outcomes (9)

• BCVA responder rate (≥ 15 letters)

  6 months

• BCVA responder rate (≥ 10 letters)

  6 months

• Peak contrast sensitivity (CS) responder rate (≥ 0.3 log CS)

  6 months

• Mean change in VA LV VFQ-48 mobility domain test scores

  6 months

• Mean change in VA LV VFQ-48 visual ability (overall) test scores

  6 months

Study Arms (2)

6.0M jCell injection

EXPERIMENTAL

Single intravitreal injection of 6.0 million retinal progenitor cells into the study eye

Biological: human retinal progenitor cells

Sham-treated control

SHAM COMPARATOR

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

Other: Mock injection

Interventions

Mock injection 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.

Sham-treated control

human retinal progenitor cells BIOLOGICAL

Single intravitreal injection of 6.0 million retinal progenitor cells (RPCs)

Also known as: jCell, famzeretcel

6.0M jCell injection

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.
• 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. If genotyping results from a certified genetic testing lab document mutations clearly diagnostic of RP, with no other potential diagnosis of a disease(s) distinct from RP, the genetic test may be used in lieu of an ERG.
• Subject age ≥ 18 years and ≤ 60 years at time of signing of consent.
• Interocular BCVA disparity ≤ 15 letters.
• Central subfield thickness (CST) ≥ 130 µm in the study eye.
• 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.
• 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.
• 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.
• Central island visual field area (central island contiguous to fixation), of ≥ 50.3 deg2 (\~ central island visual field diameter ≥ 8°) in the study eye.
• 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.
• Willingness of subject to provide a blood sample for human leukocyte antigen (HLA) typing, if not done previously with available results.
• Willingness of subject to consent to testing for RP gene mutation typing, if not performed previously with available results.
• Adequate organ function.
• Negative active infectious disease screen (active infection with Hepatitis B, C, human immunodeficiency virus \[HIV\]).
• A female subject of childbearing potential must have a negative pregnancy test (urine human chorionic gonadotropin) at entry (prior to treatment).

You may not qualify if:

• Participation in any clinical trial of a drug intervention within the last 6 months, with the exception of a N-acetyl cysteine (NAC) study.
• 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).
• Subject is currently breast feeding/pumping or is planning to breast feed/pump during the 12 months after study treatment.
• Subject is pregnant or intends to become pregnant less than 12 months after jCell injection.
• Known allergy to gentamicin.
• History of adverse reaction to dimethyl sulfoxide (DMSO).
• 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.
• 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. Examples include central serous retinopathy, vitreomacular traction, pattern/vitelliform dystrophy
• 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.
• Concurrent use of any prohibited therapies.
• 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).
• 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.
• Uncontrolled blood pressure defined as systolic pressure \> 180mmHg and/or diastolic blood pressure \> 110mmHg, while subject is at rest.
• Any chronic systemic disease requiring continuous treatment with systemic steroids or immunosuppressive agents.
• 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.

Sponsors & Collaborators

• jCyte, Inc: lead

Study Sites (14)

Associated Retina Consultants

Phoenix, Arizona, 85020, United States

RECRUITING

California Retina Consultants

Bakersfield, California, 93309, United States

RECRUITING

Retina-Vitreous Associates Medical Group

Beverly Hills, California, 90074, United States

RECRUITING

Gavin Herbert Eye Institute, UC Irvine

Irvine, California, 92697, United States

RECRUITING

Retina Consultants Medical Group

Sacramento, California, 95825, United States

RECRUITING

Bay Area Retina Associates

Walnut Creek, California, 94598, United States

RECRUITING

Vitreo Retinal Associates

Gainesville, Florida, 32607, United States

RECRUITING

Georgia Retina

Marietta, Georgia, 30060, United States

RECRUITING

Illinois Retina Associates

Oak Park, Illinois, 60304, United States

RECRUITING

NJRetina

Teaneck, New Jersey, 07666, United States

RECRUITING

Long Island Vitreoretinal Consultants

Westbury, New York, 11590, United States

RECRUITING

Retina Consultants of Texas: Bellaire Retina Center

Bellaire, Texas, 77401, United States

RECRUITING

Retina Consultants of Texas: San Antonio

San Antonio, Texas, 78240, United States

RECRUITING

The Retina Group of Washington

Fairfax, Virginia, 22031, United States

RECRUITING

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Henry Klassen, MD, PhD

  jCyte, Inc

  PRINCIPAL INVESTIGATOR

Central Study Contacts

jCyte Sr. Director of Clinical Operations

CONTACT

949-688-1816; info@jcyte.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 26, 2025
• First Posted: April 6, 2025
• Study Start: June 20, 2025
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: April 13, 2026

Record last verified: 2026-02

Locations

US (14)