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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
31
Single intravitreal injection of 6.0 million retinal progenitor cells (RPCs)
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.
Associated Retina Consultants
Phoenix, Arizona, United States
California Retina Consultants
Bakersfield, California, United States
Retina-Vitreous Associates Medical Group
Beverly Hills, California, United States
Safety of Intravitreal Injection of 6.0M jCell
Assessed by treatment emergent adverse events, immunogenicity and safety visual assessments
Time frame: 6 months
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 months
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 months
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 months
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
Mean change in VA LV VFQ-48 visual ability (overall) test scores
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Gavin Herbert Eye Institute, UC Irvine
Irvine, California, United States
Retina Consultants Medical Group
Sacramento, California, United States
Vitreo Retinal Associates
Gainesville, Florida, United States
Illinois Retina Associates
Oak Park, Illinois, United States
NJRetina
Teaneck, New Jersey, United States
Long Island Vitreoretinal Consultants
Westbury, New York, United States
Retina Consultants of Texas: Bellaire Retina Center
Bellaire, Texas, United States
...and 2 more locations
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. A fifth value, Visual Ability, is an aggregate score of the 4 scales, measured in units called logits, and is calculated for each person based on item weighting using Raasch analysis. Visual Ability is used broadly to represent changes in subject-reported outcomes from visit to visit. Higher positive values on the Visual Ability score represent better function and less impairment, whereas lower or negatives scores represent worse function or more impairment. Change in the Visual Ability 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
Mean change in central island visual field area
mean change in central island visual field area (visual field that is contiguous to fixation, excluding peripheral islands), as assessed by semi-automated kinetic perimetry, from baseline to 6-months post-treatment
Time frame: 6 months
Mean change in EQ-5D-5L test scores
The EQ-5D-5L is a generic measure of health status consisting of two parts. The first part (the descriptive system) assesses health in five dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and provides a descriptive profile that can be used to generate a health state profile. Each health state can be assigned a summary index score, ranging from less than 0 (where 0 is the value of a health state equivalent to dead; negative values representing values as worse than dead) to 1 (the value of full health), with higher scores indicating higher health utility. The second part of the questionnaire consists of a visual analogue scale (VAS) on which the patient rates his/her perceived health from 0 (the worst imaginable health) to 100 (the best imaginable health). Changes in health state index and EQ VAS scores are calculated by taking a subject's scores at 6 months and subtracting from them the baseline scores.
Time frame: 6 months
Mean change in peak contrast sensitivity (CS)
mean change in peak CS from baseline to 6-months post-treatment
Time frame: 6 months
Mean change in BCVA
mean change in best corrected visual acuity (BCVA) from baseline to 6-months post-treatment
Time frame: 6 months