Trial Outcomes & Findings for Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis Pigmentosa (NCT NCT02320812)

NCT ID: NCT02320812

Last Updated: 2019-03-05

Results Overview

proportion of subjects with treatment emergent adverse events (TEAE), related TEAE and severe TEAE

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 28 participants
• Primary outcome timeframe: 12 months
• Results posted on: 2019-03-05

Participant Flow

Participant milestones

Measure	Treated Subjects human retinal progenitor cells human retinal progenitor cells: single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
Overall Study STARTED	28
Overall Study COMPLETED	28
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis Pigmentosa

Baseline characteristics by cohort

Measure	Treated Subjects n=28 Participants human retinal progenitor cells human retinal progenitor cells: single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
Age, Continuous	49.2 years STANDARD_DEVIATION 14.7 • n=5 Participants
Sex: Female, Male Female	17 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	21 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	4 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	0 Participants n=5 Participants
Race (NIH/OMB) White	24 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
Region of Enrollment United States	28 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Population: all treated subjects

proportion of subjects with treatment emergent adverse events (TEAE), related TEAE and severe TEAE

Outcome measures

Measure	Treated Subjects n=28 Participants human retinal progenitor cells human retinal progenitor cells: single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)	1.0 Million Cells Cohort subjects who received 1.0 million hRPC	2.0 Million Cells Cohort subjects who received 2.0 million hRPC	3.0 Million Cells Cohort subjects who received 3.0 million hRPC
Number of Subjects With Adverse Events as a Measure of Safety and Tolerability Subjects with TEAE	25 Participants	—	—	—
Number of Subjects With Adverse Events as a Measure of Safety and Tolerability Subjects with related TEAE	21 Participants	—	—	—
Number of Subjects With Adverse Events as a Measure of Safety and Tolerability Subjects with grade 3 TEAE	1 Participants	—	—	—

SECONDARY outcome

Timeframe: 12 months

Population: all treated subjects

change in mean best corrected visual acuity in test eye versus untreated eye; BCVA is measured using an eye chart and is reported as the number of letters read correctly (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). A positive change from baseline indicates an improvement and a negative change from baseline indicates a worsening. Change between baseline and month 12 is reported.

Outcome measures

Measure	Treated Subjects n=8 Participants human retinal progenitor cells human retinal progenitor cells: single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)	1.0 Million Cells Cohort n=8 Participants subjects who received 1.0 million hRPC	2.0 Million Cells Cohort n=6 Participants subjects who received 2.0 million hRPC	3.0 Million Cells Cohort n=6 Participants subjects who received 3.0 million hRPC
Change in Mean Best Corrected Visual Acuity (BCVA)	1.4 number of letters read correctly Interval -3.0 to 11.0	1.0 number of letters read correctly Interval -3.0 to 11.0	4.8 number of letters read correctly Interval 0.0 to 8.0	9.0 number of letters read correctly Interval 0.0 to 12.0

Adverse Events

Treated Subjects

Serious events: 1 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Measure	Treated Subjects n=28 participants at risk human retinal progenitor cells human retinal progenitor cells: single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
Musculoskeletal and connective tissue disorders migratory pain	3.6% 1/28 • Number of events 1 • one year following single intravitreal injection of hRPC

Other adverse events

Measure	Treated Subjects n=28 participants at risk human retinal progenitor cells human retinal progenitor cells: single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
Eye disorders anterior chamber flare	17.9% 5/28 • Number of events 5 • one year following single intravitreal injection of hRPC
Eye disorders conjunctival hemorrhage	35.7% 10/28 • Number of events 10 • one year following single intravitreal injection of hRPC
Eye disorders eye pain	17.9% 5/28 • Number of events 5 • one year following single intravitreal injection of hRPC

Additional Information

Terry O'Neal, Chief Operating Officer

jCyte, Inc

Phone: 949-243-0688

Email: terry.oneal@jcyte.com

Results disclosure agreements

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