Trial Outcomes & Findings for Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (NCT NCT04604899)

NCT ID: NCT04604899

Last Updated: 2024-07-16

Results Overview

Assessed by percentage of subjects with treatment emergent adverse events

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 30 participants
• Primary outcome timeframe: 12 months
• Results posted on: 2024-07-16

Participant Flow

Participant milestones

Measure	Retreated Subjects Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. human retinal progenitor cells: single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)
Overall Study STARTED	30
Overall Study COMPLETED	23
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa

Baseline characteristics by cohort

Measure	Retreated Subjects n=30 Participants Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. human retinal progenitor cells: single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)
Age, Continuous	51.6 Years STANDARD_DEVIATION 13.93 • n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	17 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	25 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	26 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Height	170.14 cm STANDARD_DEVIATION 9.856 • n=5 Participants
Weight	84.00 kg STANDARD_DEVIATION 24.387 • n=5 Participants
Study eye Right (OD)	14 Participants n=5 Participants
Study eye Left (OS)	16 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Population: Safety population: all subjects who receive any study treatment

Assessed by percentage of subjects with treatment emergent adverse events

Outcome measures

Measure	Retreated Subjects n=30 Participants Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. human retinal progenitor cells: single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)
Safety of Intravitreal Injection of hRPC Subjects with any TEAEs	15 Participants
Safety of Intravitreal Injection of hRPC Subjects with any TEAEs related/possibly related to study drug	6 Participants
Safety of Intravitreal Injection of hRPC Subjects with any serious TEAEs	6 Participants
Safety of Intravitreal Injection of hRPC Subjects with any serious TEAEs related/possibly related to study drug	2 Participants
Safety of Intravitreal Injection of hRPC Subjects with any severe TEAEs related/possibly related to study drug	1 Participants

SECONDARY outcome

Timeframe: 12 months

Mean change in BCVA in study eye from baseline to month 12 as assessed by E-ETDRS. 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.

Outcome measures

Measure	Retreated Subjects n=23 Participants Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. human retinal progenitor cells: single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)
Best Corrected Visual Acuity (BCVA)	-2.340 letters correct Standard Deviation 5.8963

SECONDARY outcome

Timeframe: 12 months

Mean change in total kinetic visual field (KVF) area (degrees squared) of all islands of vision from baseline to 12 months. The Octopus 900 will be used for KVF testing using a specified target of V4e for subjects with a more severely impaired visual field (<10,000deg2) and a target of III4e and I4e for better seeing subjects (>10,000deg2). Target size is selected based on the Baseline visit. Whatever target size(s) is/are selected, the same size will be used throughout the study on that particular eye for that particular patient.

Outcome measures

Measure	Retreated Subjects n=23 Participants Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. human retinal progenitor cells: single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)
Kinetic Visual Field Area	-493.96 degrees squared Standard Deviation 1268.067

SECONDARY outcome

Timeframe: 12 months

Contrast sensitivity (CS) measures the ability of a subject to distinguish between finer and finer increments of light versus dark (contrast), as measured with a vertical striped pattern that varies in stripe width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each size of the target (i.e., at various CPDs). A CS curve is created by using the threshold of CS means at each target size, with the highest or most sensitive value (regardless of the CPD) representing the peak of the curve (i.e., peak contrast sensitivity). The unit of measure is therefore the peak contrast sensitivity regardless of the target size (i.e., CPD) being used to perform the measurement. The higher the value, the better the ability to detect contrast. The data shown here represent the mean change from Baseline to 12 months in the subjects' peak of the CS curve.

Outcome measures

Measure	Retreated Subjects n=16 Participants Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. human retinal progenitor cells: single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)
Contrast Sensitivity (Peak)	-0.57 Arbitrary Units (AU) Standard Deviation 6.690

SECONDARY outcome

Timeframe: 12 months

The LLMT identifies the performance of 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.

Outcome measures

Measure	Retreated Subjects n=21 Participants Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. human retinal progenitor cells: single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)
Low Luminance Mobility Test (LLMT)	-0.1 scores on a scale Standard Deviation 1.96

Adverse Events

Retreated Subjects (All Ocular AEs Were Reported in Study Eyes)

Serious events: 6 serious events

Other events: 6 other events

Deaths: 2 deaths

Serious adverse events

Measure	Retreated Subjects (All Ocular AEs Were Reported in Study Eyes) n=30 participants at risk Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. human retinal progenitor cells: single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)
Eye disorders Tractional retinal detachment	3.3% 1/30 • Number of events 1 • 12 months
Eye disorders Vitreoretinal traction syndrome	3.3% 1/30 • Number of events 1 • 12 months
Infections and infestations COVID-19	3.3% 1/30 • Number of events 1 • 12 months
Infections and infestations COVID-19 pneumonia	3.3% 1/30 • Number of events 1 • 12 months
Infections and infestations Pneumonia	3.3% 1/30 • Number of events 1 • 12 months
Cardiac disorders Acute left ventricular failure	3.3% 1/30 • Number of events 1 • 12 months
Ear and labyrinth disorders Vertigo positional	3.3% 1/30 • Number of events 1 • 12 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Non-small cell lung cancer	3.3% 1/30 • Number of events 1 • 12 months
Nervous system disorders Cerebrovascular accident	3.3% 1/30 • Number of events 2 • 12 months
Psychiatric disorders Hallucination	3.3% 1/30 • Number of events 1 • 12 months
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	3.3% 1/30 • Number of events 1 • 12 months
Respiratory, thoracic and mediastinal disorders Pleural effusion	3.3% 1/30 • Number of events 1 • 12 months
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	3.3% 1/30 • Number of events 1 • 12 months

Other adverse events

Measure	Retreated Subjects (All Ocular AEs Were Reported in Study Eyes) n=30 participants at risk Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. human retinal progenitor cells: single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)
Eye disorders Conjunctival haemorrhage	20.0% 6/30 • Number of events 6 • 12 months

Additional Information

Chief Strategy Officer

jCyte, Inc.

Phone: 9492430687

Email: media@jcyte.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place