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)
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.
⁃ 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.
⁃ 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 as assessed by semi-automated kinetic visual field (KVF) using the Octopus 900 (target size V4e), with the reaction times turned off.
‣ 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).
‣ Women of childbearing potential must agree to use a medically accepted method of contraception for at least 12 months after jCell injection.
‣ For male patients whose partners are of child-bearing potential, willingness to use a medically accepted method of contraception.