Safety of a Single, Intravitreal Injection of 6.0M jCell (Famzeretcel) in Retinitis Pigmentosa (RP)
ConditionRetinitis Pigmentosa
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-60
SponsorjCyte, Inc
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
human retinal progenitor cells
Biological/VaccineSingle intravitreal injection of 6.0 million retinal progenitor cells (RPCs)
Mock injection
Other interventionPressing 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.
Treatment groups
60 Participants
are divided into 2 treatment groupsGroup A: 6.0M jCell injectionExperimental treatment 1 intervention
Group B: Sham-treated controlSham comparator 1 intervention
Trial outcomes
Primary outcomes
1
Safety of Intravitreal Injection of 6.0M jCell
Assessed by treatment emergent adverse events, immunogenicity and safety visual assessments
Time frame
6 monthsSecondary outcomes
1
BCVA responder rate (≥ 15 letters)
proportion of responders who achieve at least 15 ETDRS letter improvement in best corrected visual acuity (BCVA) from baseline to 6-months post-treatment
Time frame
6 months2
BCVA responder rate (≥ 10 letters)
proportion of responders who achieve at least 10 ETDRS letter improvement in best corrected visual acuity (BCVA) from baseline to 6-months post-treatment
Time frame
6 months3
Peak contrast sensitivity (CS) responder rate (≥ 0.3 log CS)
proportion of responders who achieve at least a 0.3 log contrast sensitivity (CS) improvement in peak CS from baseline to 6-months post-treatment
Time frame
6 months4
Mean change in VA LV VFQ-48 mobility domain test scores
The VA LV VFQ-48 (VFQ) is used to capture changes in patients' self-reporting of their difficulty with reading, mobility and performing other daily living activities affected by visual impairment. There are 4 scales on the VFQ including Visual Information, Reading, Visual Motor, and Mobility. The data shown in this outcome measure is focused on the fourth scale, Mobility, and is measured in units called logits. Higher positive values on the Mobility score represent better function and less impairment, whereas lower or negatives scores represent worse function or more impairment. Change in the Mobility scale on the VFQ is calculated by taking a subject's score at 6 months and subtracting from it the baseline score.
Time frame
6 months Source ClinicalTrials.gov