A Phase 2 interventional study of human retinal progenitor cells and Mock injection in Retinitis Pigmentosa, sponsored by jCyte, Inc. Completed at 3 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-06-18.
Sponsored by jCyte, Inc · Phase 2, Interventional, and Treatment
This study evaluates the changes in visual function at 12 months following a single injection of human retinal progenitor cells compared to sham treated controls in a cohort of adult subjects with RP.
There is no effective treatment for RP; once photoreceptors are lost, they do not regenerate. The rate of deterioration of vision varies from person to person, with most people with RP legally blind by age 40. Preclinical studies demonstrated that transplantation of retinal progenitor cells into the eye can result in both photoreceptor replacement and significant slowing of host photoreceptor loss. Thus, the primary goal of this therapy is to preserve, and potentially improve, vision by intervening in the disease at a time when dystrophic host photoreceptors can be protected and reactivated. Based on the demonstration of acceptable safety and tolerability in a phase 1/2a study, this phase 2b study is designed as a controlled comparison of the changes in visual function and functional vision in subjects who receive a single jCell injection in comparison to a comparable sham-treated control group of subjects with RP.
273 studies on the registry are indexed under Retinitis Pigmentosa; 71 are open to participants now.
This study's enrollment of 84 is above the median of 27 across 177 interventional studies indexed under Retinitis Pigmentosa.
Browse Retinitis Pigmentosa studies →jCyte, Inc is the lead sponsor of 4 studies on the registry; none are open to participants now.
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Clinical diagnosis of RP confirmed by ERG and willing to consent to mutation typing, if not already done Best corrected visual acuity (BCVA) 20/80 or worse and no worse than 20/800 Adequate organ function and negative infectious disease screen Female of childbearing potential must have negative pregnancy test and be willing to use medically accepted methods of contraception throughout the study
Exclusion Criteria:
Eye disease other than RP that impairs visual function Pseudo-RP, cancer-associated retinopathies History of malignancy or other end-stage organ disease, or any chronic disease requiring continuous treatment with system steroids, anticoagulants or immunosuppressive agents Known allergy to penicillin or streptomycin Treatment with corticosteroids or any investigational or neuroprotectant therapy within 90 days of enrollment Cataract surgery within 3 months prior to enrollment
single intravitreal injection of 3.0 x 10e6 human retinal progenitor cells into the eye with the poorest visual acuity or, if vision is comparable in both eyes, the non-dominant eye
Biological: human retinal progenitor cells
a mock injection will be performed on the eye with the poorest vision in each Control subject (designated as the "study eye")
Other: Mock injection
single intravitreal injection of 6.0 x 10e6 human retinal progenitor cells into the eye with the poorest visual acuity or, if vision is comparable in both eyes, the non-dominant eye
Biological: human retinal progenitor cells
live suspension of 3.0 or 6.0 x 10e6 human retinal progenitor cells (hRPC) suspended in clinical grade medium injected intravitreally under local anesthesia
Also known as: jCell
pressing the hub of a syringe with no needle against the eye to mimic intravitreal injection
Best Corrected Visual Acuity (BCVA)
Mean change in BCVA in study eye from baseline to month 12 as assessed by E-ETDRS in ITT population. A letter score is used to compare change over time, with a higher number of letters representing better visual function, and a lower number of letters representing worse visual function. For example, 85 letters is equivalent to 20/20 visual acuity and 5 letters is equivalent to 20/800 visual acuity. A change value is derived for each subject by taking the letter score at 12 months and subtracting the letter score at baseline. A mean of all change values is then calculated for each arm.
Time frame: 12 months
Contrast Sensitivity (CS) at 1.0 CPD
Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. For example, if a subject's CS peak appears to be at 2.0 CPD, then additional testing occurs at 1.0 and 4.0 CPD. RP patients have suppressed CS curves and the most common pattern widths are at 0.5, 1.0, 2.0, and 4.0 CPD. Severely impaired subjects (e.g., BCVA \< 20/400) usually have flat curves with low values (e.g., 1.28), while in mildly impaired RP subjects the values can be higher (e.g., 7.12), but are rarely near normal. These data represent mean change in CS from baseline to 12 months, with greater values representing greater improvement in visual function.
Time frame: 12 months
Kinetic Visual Field (KVF)
Mean change in total area (degrees squared) of all islands of vision from baseline to 12 months.
Time frame: 12 months
Low Luminance Mobility Test (LLMT)
The LLMT identifies the performance of RP patients as they walk along an indoor pathway of arrows and obstacles at varying lighting levels. The Critical Illumination Level (CIL) is the light level below which the patient has a markedly slower pace and more errors than all light levels above (brighter than) that point. The LLMT uses light levels that go from very dim (0.12 lux) to a bright indoor room (500 lux), with evenly spaced increments that increase light by doubling the brightness of the room from the prior level. These evenly spaced light levels have been converted to a scale score to enable easier calculation of change scores. The dimmest light level of 0 lux (completely dark room) corresponds to a scale score of 13, whereas the brightest light level of 500 lux corresponds to a scale score of 0. A positive scale score change from baseline to 12 months represents improvement in low light vision, whereas a negative scale score change represents a decline in low light vision.
Time frame: 12 months
Low Vision Functional Questionnaire (Visual Ability)
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 12 months and subtracting from it the baseline score.
Time frame: 12 months
Safety of Intravitreal Injection of Retinal Progenitor Cells (RPC)
Assessed by treatment emergent adverse events, immunogenicity and safety visual assessments
Time frame: 12 months
85 subjects were enrolled; 84 subjects were randomized
| Milestone | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Started | 29 | 27 | 27 |
| Completed | 28 | 26 | 26 |
| Not completed | 1 | 1 | 1 |
| Withdrew: Lost to follow-up | 1 | 0 | 0 |
| Withdrew: Physician decision | 0 | 1 | 0 |
| Withdrew: Withdrawal by subject | 0 | 0 | 1 |
Mean change in BCVA in study eye from baseline to month 12 as assessed by E-ETDRS in ITT population. A letter score is used to compare change over time, with a higher number of letters representing better visual function, and a lower number of letters representing worse visual function. For example, 85 letters is equivalent to 20/20 visual acuity and 5 letters is equivalent to 20/800 visual acuity. A change value is derived for each subject by taking the letter score at 12 months and subtracting the letter score at baseline. A mean of all change values is then calculated for each arm.
| Letters correct | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Best Corrected Visual Acuity (BCVA) | 3.9 ± 9.92 | 1.3 ± 14.14 | 2.6 ± 21.53 |
Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. For example, if a subject's CS peak appears to be at 2.0 CPD, then additional testing occurs at 1.0 and 4.0 CPD. RP patients have suppressed CS curves and the most common pattern widths are at 0.5, 1.0, 2.0, and 4.0 CPD. Severely impaired subjects (e.g., BCVA \< 20/400) usually have flat curves with low values (e.g., 1.28), while in mildly impaired RP subjects the values can be higher (e.g., 7.12), but are rarely near normal. These data represent mean change in CS from baseline to 12 months, with greater values representing greater improvement in visual function.
| CS at 1.0 cycles per degree (CPD) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Contrast Sensitivity (CS) at 1.0 CPD | 0.4331 ± 1.5011 | 1.9145 ± 4.3072 | 0.1142 ± 4.9526 |
Mean change in total area (degrees squared) of all islands of vision from baseline to 12 months.
| Total area (degrees squared) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Kinetic Visual Field (KVF) | 260.19 ± 900.024 | -44.02 ± 1001.614 | 560.19 ± 2453.259 |
The LLMT identifies the performance of RP patients as they walk along an indoor pathway of arrows and obstacles at varying lighting levels. The Critical Illumination Level (CIL) is the light level below which the patient has a markedly slower pace and more errors than all light levels above (brighter than) that point. The LLMT uses light levels that go from very dim (0.12 lux) to a bright indoor room (500 lux), with evenly spaced increments that increase light by doubling the brightness of the room from the prior level. These evenly spaced light levels have been converted to a scale score to enable easier calculation of change scores. The dimmest light level of 0 lux (completely dark room) corresponds to a scale score of 13, whereas the brightest light level of 500 lux corresponds to a scale score of 0. A positive scale score change from baseline to 12 months represents improvement in low light vision, whereas a negative scale score change represents a decline in low light vision.
| Scores on a scale | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Low Luminance Mobility Test (LLMT) | 0.6 ± 2.00 | -0.3 ± 2.49 | 0.2 ± 1.96 |
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 12 months and subtracting from it the baseline score.
| logits | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Low Vision Functional Questionnaire (Visual Ability) | 0.225 ± 0.4194 | 0.146 ± 0.5228 | 0.360 ± 0.6963 |
Assessed by treatment emergent adverse events, immunogenicity and safety visual assessments
| participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Subjects with TEAE | 15 | 20 | 21 |
| Subjects with related/possibly related TEAE | 7 | 14 | 13 |
| Subjects with severe TEAE | 0 | 3 | 1 |
| Subjects with SAE | 0 | 1 | 0 |
| Subjects with CME absent at baseline and present at M12 (OCT) | 1 | 0 | 0 |
| Subjects with clinically significant abnormal conjunctiva absent at baseline and present at M12 | 0 | 2 | 0 |
| Subjects with clinically significant abnormal sclera absent at baseline and present at M12 | 0 | 1 | 0 |
| Subjects with clinically significant abnormal AC flare absent at baseline and present at M12 | 0 | 0 | 0 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population (N=76), with a responder being defined as someone who gains at least 10 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. The PP population excludes seven subjects with major protocol violations, including two subjects who received a different treatment than the group to which they were randomized, four subjects with preexisting ophthalmic conditions meeting exclusion criteria (three glaucoma and one amblyopia), and one subject who experienced an intraocular lens subluxation (i.e., the displacement of a replacement lens following previous cataract surgery - unrelated to study drug) and could not perform endpoint testing at 12 months.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥10 Letters): Best Corrected Visual Acuity (BCVA) - PP Population | 5 | 4 | 9 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population (N=76), with a responder being defined as someone who gains at least 13 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. The PP population excludes seven subjects with major protocol violations, including two subjects who received a different treatment than the group to which they were randomized, four subjects with preexisting ophthalmic conditions meeting exclusion criteria (three glaucoma and one amblyopia), and one subject who experienced an intraocular lens subluxation (i.e., the displacement of a replacement lens following previous cataract surgery - unrelated to study drug) and could not perform endpoint testing at 12 months.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥13 Letters): Best Corrected Visual Acuity (BCVA) - PP Population | 3 | 4 | 7 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population (N=76), with a responder being defined as someone who gains at least 15 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. The PP population excludes seven subjects with major protocol violations, including two subjects who received a different treatment than the group to which they were randomized, four subjects with preexisting ophthalmic conditions meeting exclusion criteria (three glaucoma and one amblyopia), and one subject who experienced an intraocular lens subluxation (i.e., the displacement of a replacement lens following previous cataract surgery - unrelated to study drug) and could not perform endpoint testing at 12 months.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥15 Letters): Best Corrected Visual Acuity (BCVA) - PP Population | 2 | 3 | 6 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population (N=76), with a responder being defined as someone who gains at least 20 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. The PP population excludes seven subjects with major protocol violations, including two subjects who received a different treatment than the group to which they were randomized, four subjects with preexisting ophthalmic conditions meeting exclusion criteria (three glaucoma and one amblyopia), and one subject who experienced an intraocular lens subluxation (i.e., the displacement of a replacement lens following previous cataract surgery - unrelated to study drug) and could not perform endpoint testing at 12 months.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥20 Letters): Best Corrected Visual Acuity (BCVA) - PP Population | 1 | 2 | 4 |
Mean change in BCVA in study eye from baseline to month 12 as assessed by E-ETDRS in the Per Protocol (PP) population (N=76). The PP population excludes seven subjects with major protocol violations, including two subjects who received a different treatment than the group to which they were randomized, four subjects with preexisting ophthalmic conditions meeting exclusion criteria (three glaucoma and one amblyopia), and one subject who experienced an intraocular lens subluxation (i.e., the displacement of a replacement lens following previous cataract surgery - unrelated to study drug) and could not perform endpoint testing at 12 months. Refer to Outcome Measure #1 for more information on how change in BCVA from baseline to month 12 is assessed.
| Letters correct | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Best Corrected Visual Acuity (BCVA) - PP Population | 2.8 ± 8.69 | 3.0 ± 11.43 | 7.4 ± 15.57 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population (N=76), with a responder being defined as someone who gains at least 100% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to month 12 is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥100%): Peak Contrast Sensitivity (CS) - PP Population | 1 | 2 | 5 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population (N=76), with a responder being defined as someone who gains at least 150% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to month 12 is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥150%): Peak Contrast Sensitivity (CS) - PP Population | 1 | 0 | 4 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population (N=76), with a responder being defined as someone who gains at least 200% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to month 12 is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥200%): Peak Contrast Sensitivity (CS) - PP Population | 1 | 0 | 2 |
Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. The data shown here are for mean change from baseline to 12 months in the peak of the CS curve in the Per Protocol (PP) population (N=76). Refer to Outcome Measure #7 for background information on the PP population. Refer to Outcome Measure #2 for more information on how change in CS from baseline to month 12 is assessed.
| Peak CS | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Peak Contrast Sensitivity (CS) - PP Population | 1.3454 ± 4.4164 | 0.1294 ± 2.4379 | 3.1329 ± 12.4076 |
Mean change in total area (degrees squared) of all islands of vision from baseline to 12 months in the Per Protocol (PP) Population (N=76). Refer to Outcome Measure #7 for background information on the PP population.
| Total area (degrees squared) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Kinetic Visual Field (KVF) - PP Population | 229.13 ± 908.525 | -37.81 ± 1021.757 | 594.47 ± 2558.858 |
The LLMT identifies the performance of RP patients as they walk along an indoor pathway of arrows and obstacles at varying lighting levels. The Critical Illumination Level (CIL) is the light level below which the patient has a markedly slower pace and more errors than all light levels above (brighter than) that point. The LLMT uses light levels that go from very dim (0.12 lux) to a very bright (500 lux), with evenly spaced increments that increase light by doubling the brightness of the room from the prior level. These evenly spaced light levels have been converted to a scale score to enable easier calculation of change scores. The dimmest level of 0 lux corresponds to a scale score of 13, whereas the brightest level of 500 lux corresponds to a scale score of 0. A positive scale score change from baseline to 12 months represents improvement, whereas a negative scale score change represents a decline. Refer to Outcome Measure #7 for background information on the PP population.
| Scores on a scale | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Low Luminance Mobility Test (LLMT) - PP Population | 0.6 ± 2.06 | 0.4 ± 1.14 | 0.4 ± 1.82 |
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 12 months and subtracting from it the baseline score. Refer to Outcome Measure #7 for background information on the PP population.
| logits | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Low Vision Functional Questionnaire (Visual Ability) - PP Population | 0.238 ± 0.4324 | 0.151 ± 0.5329 | 0.371 ± 0.7335 |
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 12 months and subtracting from it the baseline score.
| logits | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Low Vision Functional Questionnaire (Mobility) - PP Population | 0.024 ± 0.499 | 0.256 ± 0.716 | 0.521 ± 0.813 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline (N=55), with a responder being defined as someone who gains at least 10 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. The work undertaken to understand what contributed to variability in measurements in the Phase 2b study, combined with the available published literature, has led to the identification of the characteristics of individuals who provide inconsistent/unreliable data. One such characteristic is found in subjects in whom there is a substantial BCVA disparity of more than 15 letters between their study (worse) eye and fellow (better) eye. This disparity has the potential to lead to variable testing results due to the brain's un-suppression of the image from their worse-seeing eye under clinical study monocular test conditions where their dominant eye is covered.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥10 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes | 2 | 2 | 8 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline (N=55), with a responder being defined as someone who gains at least 13 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. The work undertaken to understand what contributed to variability in measurements in the Phase 2b study, combined with the available published literature, has led to the identification of the characteristics of individuals who provide inconsistent/unreliable data. One such characteristic is found in subjects in whom there is a substantial BCVA disparity of more than 15 letters between their study (worse) eye and fellow (better) eye. This disparity has the potential to lead to variable testing results due to the brain's un-suppression of the image from their worse-seeing eye under clinical study monocular test conditions where their dominant eye is covered.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥13 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes | 1 | 2 | 6 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline (N=55), with a responder being defined as someone who gains at least 15 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. The work undertaken to understand what contributed to variability in measurements in the Phase 2b study, combined with the available published literature, has led to the identification of the characteristics of individuals who provide inconsistent/unreliable data. One such characteristic is found in subjects in whom there is a substantial BCVA disparity of more than 15 letters between their study (worse) eye and fellow (better) eye. This disparity has the potential to lead to variable testing results due to the brain's un-suppression of the image from their worse-seeing eye under clinical study monocular test conditions where their dominant eye is covered.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥15 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes | 0 | 1 | 5 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline (N=55), with a responder being defined as someone who gains at least 20 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. The work undertaken to understand what contributed to variability in measurements in the Phase 2b study, combined with the available published literature, has led to the identification of the characteristics of individuals who provide inconsistent/unreliable data. One such characteristic is found in subjects in whom there is a substantial BCVA disparity of more than 15 letters between their study (worse) eye and fellow (better) eye. This disparity has the potential to lead to variable testing results due to the brain's un-suppression of the image from their worse-seeing eye under clinical study monocular test conditions where their dominant eye is covered.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥20 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes | 0 | 0 | 3 |
Mean change in BCVA from Baseline to month 12 as assessed by E-ETDRS in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline (N=55). The work undertaken to understand what contributed to variability in measurements in the Phase 2b study, combined with the available published literature, has led to the identification of the characteristics of individuals who provide inconsistent/unreliable data. One such characteristic is found in subjects in whom there is a substantial BCVA disparity of more than 15 letters between their study (worse) eye and fellow (better) eye. This disparity has the potential to lead to variable testing results due to the brain's un-suppression of the image from their worse-seeing eye under clinical study monocular test conditions where their dominant eye is covered. Refer to Outcome Measure #1 for more information on how change in BCVA from baseline to month 12 is assessed.
| Letters correct | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes | 0.86 ± 6.54 | 0.82 ± 7.98 | 10.25 ± 15.91 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline (N=55), with a responder being defined as someone who gains at least 100% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥100%): Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes | 0 | 2 | 5 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline (N=55), with a responder being defined as someone who gains at least 150% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥150%): Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes | 0 | 0 | 4 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline (N=55), with a responder being defined as someone who gains at least 200% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥200%): Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes | 0 | 0 | 2 |
Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. The data shown here are for mean change from baseline to 12 months in the peak of the CS curve in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline (N=55). Refer to Outcome Measure #12 for background information on this analysis population. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Peak CS | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes | 0.61 ± 1.85 | 0.11 ± 2.76 | 6.09 ± 13.87 |
Mean change in total area (degrees squared) of all islands of vision from baseline to 12 months in the Per Protocol (PP) Population with a study eye that is ≤15 letters worse than the fellow eye at baseline (N=55). Refer to Outcome Measure #12 for background information on this analysis population.
| Total area (degrees squared) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Kinetic Visual Field (KVF) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes | 313.33 ± 964.08 | -173.46 ± 1027.19 | 843.93 ± 3035.82 |
The LLMT identifies the performance of RP patients as they walk along an indoor pathway of arrows and obstacles at varying lighting levels. The Critical Illumination Level (CIL) is the light level below which the patient has a markedly slower pace and more errors than all light levels above (brighter than) that point. The LLMT uses light levels that go from very dim (0.12 lux) to very bright (500 lux), with evenly spaced increments that increase light by doubling the brightness of the room from the prior level. These evenly spaced light levels have been converted to a scale score to enable easier calculation of change scores. The dimmest level of 0 lux corresponds to a scale score of 13, whereas the brightest level of 500 lux corresponds to a scale score of 0. A positive scale score change from baseline to 12 months represents improvement, whereas a negative scale score change represents a decline. Refer to Outcome Measure #12 for background information on this analysis population.
| scores on a scale | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Low Luminance Mobility Test (LLMT) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes | 0.82 ± 2.24 | 0.43 ± 1.22 | 0.80 ± 1.93 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye and with the ability to maintain fixation at baseline (N=42), with a responder being defined as someone who gains at least 10 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. Beyond the BCVA disparity characteristic described in the previous post-hoc outcome measures, another characteristic that was found to lead to high degrees of testing variability was the inability to maintain fixation as assessed by a fixation score of 1 on kinetic visual field testing. This inability was due to either an atypical form of RP which caused the development of a central scotoma (blind spot), or the absence of remaining central visual field. These individuals tended to have extremely variable results that were dependent upon their ability to use unsteady "peripheral" eccentric viewing to perform the clinical endpoint tests.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥10 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | 1 | 1 | 8 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye and with the ability to maintain fixation at baseline (N=42), with a responder being defined as someone who gains at least 13 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. Beyond the BCVA disparity characteristic described in the previous post-hoc outcome measures, another characteristic that was found to lead to high degrees of testing variability was the inability to maintain fixation as assessed by a fixation score of 1 on kinetic visual field testing. This inability was due to either an atypical form of RP which caused the development of a central scotoma (blind spot), or the absence of remaining central visual field. These individuals tended to have extremely variable results that were dependent upon their ability to use unsteady "peripheral" eccentric viewing to perform the clinical endpoint tests.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥13 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | 0 | 1 | 6 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye and with the ability to maintain fixation at baseline (N=42), with a responder being defined as someone who gains at least 15 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. Beyond the BCVA disparity characteristic described in the previous post-hoc outcome measures, another characteristic that was found to lead to high degrees of testing variability was the inability to maintain fixation as assessed by a fixation score of 1 on kinetic visual field testing. This inability was due to either an atypical form of RP which caused the development of a central scotoma (blind spot), or the absence of remaining central visual field. These individuals tended to have extremely variable results that were dependent upon their ability to use unsteady "peripheral" eccentric viewing to perform the clinical endpoint tests.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥15 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | 0 | 1 | 5 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye and with the ability to maintain fixation at baseline (N=42), with a responder being defined as someone who gains at least 20 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. Beyond the BCVA disparity characteristic described in the previous post-hoc outcome measures, another characteristic that was found to lead to high degrees of testing variability was the inability to maintain fixation as assessed by a fixation score of 1 on kinetic visual field testing. This inability was due to either an atypical form of RP which caused the development of a central scotoma (blind spot), or the absence of remaining central visual field. These individuals tended to have extremely variable results that were dependent upon their ability to use unsteady "peripheral" eccentric viewing to perform the clinical endpoint tests.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥20 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | 0 | 0 | 3 |
Mean change in BCVA in study eye from baseline to month 12 as assessed by E-ETDRS in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye and with the ability to maintain fixation at baseline (N=42). Beyond the BCVA disparity characteristic described in the previous post-hoc outcome measures, another characteristic that was found to lead to high degrees of testing variability was the inability to maintain fixation as assessed by a fixation score of 1 on kinetic visual field testing. This inability was due to either an atypical form of RP which caused the development of a central scotoma (blind spot), or the absence of remaining central visual field. These individuals tended to have extremely variable results that were dependent upon their ability to use unsteady "peripheral" eccentric viewing to perform the clinical endpoint tests. Refer to Outcome Measure #1 for more information on how change in BCVA from baseline to month 12 is assessed.
| Letters correct | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | 2.00 ± 5.75 | 0.29 ± 7.56 | 13.00 ± 16.47 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline and with the ability to maintain fixation at Baseline (N=42), with a responder being defined as someone who gains at least 100% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥100%): Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | 0 | 2 | 5 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline and with the ability to maintain fixation at Baseline (N=42), with a responder being defined as someone who gains at least 150% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥150%): Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | 0 | 0 | 4 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye at baseline and with the ability to maintain fixation at Baseline (N=42), with a responder being defined as someone who gains at least 200% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥200%): Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | 0 | 0 | 2 |
Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. The data shown here are for mean change from baseline to 12 months in the peak of the CS curve in the PP Population with a study eye that is ≤15 letters worse than the fellow eye and with the ability to maintain fixation at Baseline (N=42). Refer to Outcome Measure #16 for background information on this analysis population. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Peak CS | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | 0.812 ± 2.049 | 0.020 ± 2.877 | 7.089 ± 14.703 |
Mean change in total area (degrees squared) of all islands of vision from baseline to 12 months in the Per Protocol (PP) Population with a study eye that is ≤15 letters worse than the fellow eye and with the ability to maintain fixation at baseline (N=42). Refer to Outcome Measure #16 for background information on this analysis population.
| Total area (degrees squared) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Kinetic Visual Field (KVF) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | 135.14 ± 595.55 | -94.83 ± 1039.37 | 1263.68 ± 3208.76 |
The LLMT identifies the performance of RP patients as they walk along an indoor pathway of arrows and obstacles at varying lighting levels. The Critical Illumination Level (CIL) is the light level below which the patient has a markedly slower pace and more errors than all light levels above (brighter than) that point. The LLMT uses light levels that go from very dim (0.12 lux) to very bright (500 lux), with evenly spaced increments that increase light by doubling the brightness of the room from the prior level. These evenly spaced light levels have been converted to a scale score to enable easier calculation of change scores. The dimmest level of 0 lux corresponds to a scale score of 13, whereas the brightest level of 500 lux corresponds to a scale score of 0. A positive scale score change from baseline to 12 months represents improvement, whereas a negative scale score change represents a decline. Refer to Outcome Measure #16 for background information on this analysis population.
| scores on a scale | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Low Luminance Mobility Test (LLMT) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | 0.33 ± 0.78 | 0.23 ± 1.01 | 0.92 ± 2.02 |
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 12 months and subtracting from it the baseline score.
| logits | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Low Vision Functional Questionnaire (Mobility) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes & Ability to Maintain Fixation | -0.054 ± 0.412 | 0.237 ± 0.306 | 0.676 ± 0.957 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye, the ability to maintain fixation, and with at least 130µm of central foveal thickness (as measured in the central subfield region) remaining at baseline (N=21), with a responder being defined as someone who gains at least 10 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. Central foveal thickness, as measured by optical coherence tomography (OCT), is believed to be an anatomical marker representing the number of surviving foveal cones. A thicker central fovea corresponds to more surviving cone photoreceptors which are available to be up-regulated.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥10 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes, Ability to Maintain Fixation, and >130µm Central Foveal Thickness | 1 | 1 | 6 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye, the ability to maintain fixation, and with at least 130µm of central foveal thickness (as measured in the central subfield region) remaining at baseline (N=21), with a responder being defined as someone who gains at least 13 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. Central foveal thickness, as measured by optical coherence tomography (OCT), is believed to be an anatomical marker representing the number of surviving foveal cones. A thicker central fovea corresponds to more surviving cone photoreceptors which are available to be up-regulated.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥13 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes, Ability to Maintain Fixation, and >130µm Central Foveal Thickness | 0 | 1 | 6 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye, the ability to maintain fixation, and with at least 130µm of central foveal thickness (as measured in the central subfield region) remaining at baseline (N=21), with a responder being defined as someone who gains at least 15 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. Central foveal thickness, as measured by optical coherence tomography (OCT), is believed to be an anatomical marker representing the number of surviving foveal cones. A thicker central fovea corresponds to more surviving cone photoreceptors which are available to be up-regulated.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥15 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes, Ability to Maintain Fixation, and >130µm Central Foveal Thickness | 0 | 1 | 5 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye, the ability to maintain fixation, and with at least 130µm of central foveal thickness (as measured in the central subfield region) remaining at baseline (N=21), with a responder being defined as someone who gains at least 20 letters in BCVA (assessed by E-ETDRS) from baseline to month 12. Central foveal thickness, as measured by optical coherence tomography (OCT), is believed to be an anatomical marker representing the number of surviving foveal cones. A thicker central fovea corresponds to more surviving cone photoreceptors which are available to be up-regulated.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥20 Letters): Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes, Ability to Maintain Fixation, and >130µm Central Foveal Thickness | 0 | 0 | 3 |
Mean change in BCVA in study eye from baseline to month 12 as assessed by E-ETDRS in the Per Protocol (PP) population with a study eye that is ≤15 letters worse than the fellow eye, the ability to maintain fixation, and with at least 130µm of central foveal thickness (as measured in the central subfield region) remaining at baseline (N=21). Central foveal thickness, as measured by optical coherence tomography (OCT), is believed to be an anatomical marker representing the number of surviving foveal cones. A thicker central fovea corresponds to more surviving cone photoreceptors which are available to be up-regulated. Refer to Outcome Measure #1 for more information on how change in BCVA from baseline to month 12 is assessed.
| Letters correct | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Best Corrected Visual Acuity (BCVA) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes, Ability to Maintain Fixation, and >130µm Central Foveal Thickness | 3.50 ± 3.96 | -0.40 ± 10.36 | 18.38 ± 18.68 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) Population with a study eye that is ≤15 letters worse than the fellow eye, the ability to maintain fixation, and with at least 130µm of central foveal thickness (as measured in the central subfield region) remaining at baseline (N=21), with a responder being defined as someone who gains at least 100% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥100%): Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes, Ability to Maintain Fixation, and >130µm Central Foveal Thickness | 0 | 0 | 3 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) Population with a study eye that is ≤15 letters worse than the fellow eye, the ability to maintain fixation, and with at least 130µm of central foveal thickness (as measured in the central subfield region) remaining at baseline (N=21), with a responder being defined as someone who gains at least 150% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥150%): Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes, Ability to Maintain Fixation, and >130µm Central Foveal Thickness | 0 | 0 | 3 |
Comparison of responder rates across treatment groups in the Per Protocol (PP) Population with a study eye that is ≤15 letters worse than the fellow eye, the ability to maintain fixation, and with at least 130µm of central foveal thickness (as measured in the central subfield region) remaining at baseline (N=21), with a responder being defined as someone who gains at least 200% in Peak CS from baseline to month 12. Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Participants | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Responder Analysis (≥200%): Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes, Ability to Maintain Fixation, and >130µm Central Foveal Thickness | 0 | 0 | 1 |
Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. The data shown here are for mean change from baseline to 12 months in the peak of the CS curve in the Per Protocol (PP) Population with a study eye that is ≤15 letters worse than the fellow eye, the ability to maintain fixation, and with at least 130µm of central foveal thickness (as measured in the central subfield region) remaining at baseline (N=21). Refer to Outcome Measure #24 for background information on this analysis population. Refer to Outcome Measure #2 for more information on how change in CS from baseline to 12 months is assessed.
| Peak CS | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Peak Contrast Sensitivity (CS) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes, Ability to Maintain Fixation, and >130µm Central Foveal Thickness | 0.777 ± 0.732 | -1.343 ± 2.492 | 10.791 ± 18.018 |
Mean change in total area (degrees squared) of all islands of vision from baseline to 12 months in the Per Protocol (PP) Population with a study eye that is ≤15 letters worse than the fellow eye, the ability to maintain fixation, and with at least 130µm of central foveal thickness (as measured in the central subfield region) remaining at bbaseline (N=21). Refer to Outcome Measure #24 for background information on this analysis population.
| Total area (degrees squared) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Kinetic Visual Field (KVF) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes, Ability to Maintain Fixation, and >130µm Central Foveal Thickness | 393.93 ± 613.69 | 107.50 ± 873.65 | 2251.86 ± 3668.83 |
The LLMT identifies the performance of RP patients as they walk along an indoor pathway of arrows and obstacles at varying lighting levels. The Critical Illumination Level (CIL) is the light level below which the patient has a markedly slower pace and more errors than all light levels above (brighter than) that point. The LLMT uses light levels that go from very dim (0.12 lux) to very bright (500 lux), with evenly spaced increments that increase light by doubling the brightness of the room from the prior level. These evenly spaced light levels have been converted to a scale score to enable easier calculation of change scores. The dimmest level of 0 lux corresponds to a scale score of 13, whereas the brightest level of 500 lux corresponds to a scale score of 0. A positive scale score change from baseline to 12 months represents improvement, whereas a negative scale score change represents a decline. Refer to Outcome Measure #24 for background information on this analysis population.
| scores on a scale | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Low Luminance Mobility Test (LLMT) - PP Population With ≤15 Letter Baseline BCVA Disparity Between Eyes, Ability to Maintain Fixation, and >130µm Central Foveal Thickness | 0.43 ± 0.98 | -0.60 ± 0.55 | 1.50 ± 2.07 |
Collected over one year. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Sham Treated Control | 0/29 (0%) | 0/29 (0%) | 13/29 (44.8%) |
| Test (jCell Injection) Dose Level 1 | 0/27 (0%) | 1/27 (3.7%) | 18/27 (66.7%) |
| Test (jCell Injection) Dose Level 2 | 0/27 (0%) | 0/27 (0%) | 14/27 (51.9%) |
| Event | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| ocular hypertensionEye disorders | 0/29 | 1/27 | 0/27 |
| Event | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 |
|---|---|---|---|
| Conjunctival hemorrhageEye disorders | 5/29 | 9/27 | 6/27 |
| Eye painEye disorders | 0/29 | 5/27 | 1/27 |
| Visual acuity reducedEye disorders | 3/29 | 4/27 | 3/27 |
| Intraocular pressure increasedInvestigations | 1/29 | 4/27 | 1/27 |
| Anterior chamber flareEye disorders | 0/29 | 0/27 | 3/27 |
| ChalazionEye disorders | 0/29 | 2/27 | 0/27 |
| Cystoid macular edemaEye disorders | 1/29 | 2/27 | 0/27 |
| Eye irritationEye disorders | 0/29 | 1/27 | 2/27 |
| SinusitisInfections and infestations | 1/29 | 1/27 | 2/27 |
| Urinary tract infectionInfections and infestations | 0/29 | 2/27 | 0/27 |
One subject received 4M dose prior to major protocol amendment that excluded this dose group; this subject is not included in the presented analyses
| Age, Categorical(Participants) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 | Total |
|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 23 | 24 | 24 | 71 |
| >=65 years | 6 | 3 | 3 | 12 |
| Sex: Female, Male(Participants) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 | Total |
|---|---|---|---|---|
| Female | 7 | 15 | 13 | 35 |
| Male | 22 | 12 | 14 | 48 |
| Ethnicity (NIH/OMB)(Participants) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 | Total |
|---|---|---|---|---|
| Hispanic or Latino | 5 | 4 | 7 | 16 |
| Not Hispanic or Latino | 24 | 23 | 20 | 67 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 6 | 2 | 5 | 13 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 1 | 2 | 1 | 4 |
| White | 22 | 23 | 20 | 65 |
| More than one race | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 1 | 1 |
| Region of Enrollment(participants) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 | Total |
|---|---|---|---|---|
| United States | 29 | 27 | 27 | 83 |
| Selection of study eye(Participants) | Sham Treated Control | Test (jCell Injection) Dose Level 1 | Test (jCell Injection) Dose Level 2 | Total |
|---|---|---|---|---|
| study eye OD | 12 | 12 | 14 | 38 |
| study eye OS | 17 | 15 | 13 | 45 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: No
Ran from Mar 2017 to primary completion in Nov 2020, 44 months.
Results were first posted in Jan 2022, 1 year 2 months after primary completion.
Of 15 phase 2 interventional studies under Retinitis Pigmentosa, half reached primary completion within 28 months of starting (the median), counting those still running; a quarter within 19 and three quarters within 41. This study's 44 months is above that.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jun 2024. You cannot join it, but the record below documents what was studied.
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