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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 6.0M jCell injection | Experimental | Single intravitreal injection of 6.0 million retinal progenitor cells into the study eye |
|
| Sham-treated control | Sham Comparator | A mock injection will be performed on the study eye in each control subject |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| human retinal progenitor cells | Biological | Single intravitreal injection of 6.0 million retinal progenitor cells (RPCs) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety of Intravitreal Injection of 6.0M jCell | Assessed by treatment emergent adverse events, immunogenicity and safety visual assessments | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 6 months |
| BCVA responder rate (≥ 10 letters) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| jCyte Sr. Director of Clinical Operations | Contact | 949-688-1816 | info@jcyte.com |
| Name | Affiliation | Role |
|---|---|---|
| Henry Klassen, MD, PhD | jCyte, Inc | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Associated Retina Consultants | Recruiting | Phoenix | Arizona | 85020 | United States |
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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.
| 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. |
|
proportion of responders who achieve at least 10 ETDRS letter improvement in best corrected visual acuity (BCVA) from baseline to 6-months post-treatment
| 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 | 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. | 6 months |
| Mean change in VA LV VFQ-48 visual ability (overall) 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. 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. | 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 | 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. | 6 months |
| Mean change in peak contrast sensitivity (CS) | mean change in peak CS from baseline to 6-months post-treatment | 6 months |
| Mean change in BCVA | mean change in best corrected visual acuity (BCVA) from baseline to 6-months post-treatment | 6 months |
| California Retina Consultants | Recruiting | Bakersfield | California | 93309 | United States |
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| Retina-Vitreous Associates Medical Group | Recruiting | Beverly Hills | California | 90074 | United States |
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| Gavin Herbert Eye Institute, UC Irvine | Recruiting | Irvine | California | 92697 | United States |
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| Retina Consultants Medical Group | Recruiting | Sacramento | California | 95825 | United States |
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| Bay Area Retina Associates | Recruiting | Walnut Creek | California | 94598 | United States |
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| Vitreo Retinal Associates | Recruiting | Gainesville | Florida | 32607 | United States |
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| Georgia Retina | Recruiting | Marietta | Georgia | 30060 | United States |
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| Illinois Retina Associates | Recruiting | Oak Park | Illinois | 60304 | United States |
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| NJRetina | Recruiting | Teaneck | New Jersey | 07666 | United States |
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| Long Island Vitreoretinal Consultants | Recruiting | Westbury | New York | 11590 | United States |
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| Retina Consultants of Texas: Bellaire Retina Center | Recruiting | Bellaire | Texas | 77401 | United States |
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| Retina Consultants of Texas: San Antonio | Recruiting | San Antonio | Texas | 78240 | United States |
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| The Retina Group of Washington | Recruiting | Fairfax | Virginia | 22031 | United States |
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| ID | Term |
|---|---|
| D012174 | Retinitis Pigmentosa |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D058499 | Retinal Dystrophies |
| D012162 | Retinal Degeneration |
| D012164 | Retinal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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