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

• ClinicalTrials.gov Identifier: NCT02320812
• Recruitment Status: Completed
• First Posted: December 19, 2014
• Results First Posted: March 5, 2019
• Last Update Posted: March 5, 2019
• Sponsor: jCyte, Inc
• Collaborator: California Institute for Regenerative Medicine (CIRM)
• Information provided by (Responsible Party): jCyte, Inc

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Retinitis Pigmentosa (RP)
• Intervention: Biological: human retinal progenitor cells
• Enrollment: 28

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Treated Subjects
Arm/Group Description	human retinal progenitor cells human retinal progenitor cells: single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
Period Title: Overall Study
Started	28
Completed	28
Not Completed	0

Baseline Characteristics

Arm/Group Title		Treated Subjects
Arm/Group Description		human retinal progenitor cells human retinal progenitor cells: single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
Overall Number of Baseline Participants		28
Baseline Analysis Population Description		[Not Specified]
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	28 participants
		49.2 (14.7)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	28 participants
	Female	17 60.7%
	Male	11 39.3%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	28 participants
	Hispanic or Latino	7 25.0%
	Not Hispanic or Latino	21 75.0%
	Unknown or Not Reported	0 0.0%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	28 participants
	American Indian or Alaska Native	0 0.0%
	Asian	4 14.3%
	Native Hawaiian or Other Pacific Islander	0 0.0%
	Black or African American	0 0.0%
	White	24 85.7%
	More than one race	0 0.0%
	Unknown or Not Reported	0 0.0%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
United States	Number Analyzed	28 participants
		28

Outcome Measures

1. Primary Outcome

Title	Number of Subjects With Adverse Events as a Measure of Safety and Tolerability
Description	proportion of subjects with treatment emergent adverse events (TEAE), related TEAE and severe TEAE
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

all treated subjects

Arm/Group Title	Treated Subjects
Arm/Group Description:	human retinal progenitor cells human retinal progenitor cells: single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
Overall Number of Participants Analyzed	28
Measure Type: Count of Participants; Unit of Measure: Participants
Subjects with TEAE	25 89.3%
Subjects with related TEAE	21 75.0%
Subjects with grade 3 TEAE	1 3.6%

2. Secondary Outcome

Title	Change in Mean Best Corrected Visual Acuity (BCVA)
Description	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.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

all treated subjects

Arm/Group Title	0.5 Million Cells Cohort	1.0 Million Cells Cohort	2.0 Million Cells Cohort	3.0 Million Cells Cohort
Arm/Group Description:	subjects who received 0.5 million human retinal progenitor cells (hRPC)	subjects who received 1.0 million hRPC	subjects who received 2.0 million hRPC	subjects who received 3.0 million hRPC
Overall Number of Participants Analyzed	8	8	6	6
Mean (Full Range); Unit of Measure: number of letters read correctly
	1.4; (-3 to 11)	1.0; (-3 to 11)	4.8; (0 to 8)	9.0; (0 to 12)

Adverse Events

Time Frame	one year following single intravitreal injection of hRPC
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Treated Subjects
Arm/Group Description	human retinal progenitor cells human retinal progenitor cells: single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
All-Cause Mortality
	Treated Subjects
	Affected / at Risk (%)
Total	0/28 (0.00%)
Serious Adverse Events
	Treated Subjects
	Affected / at Risk (%)	# Events
Total	1/28 (3.57%)
Musculoskeletal and connective tissue disorders
migratory pain † 1 [1]	1/28 (3.57%)	1
1 Term from vocabulary, MedDRA; † Indicates events were collected by systematic assessment; [1] Grade 2
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Treated Subjects
	Affected / at Risk (%)	# Events
Total	20/28 (71.43%)
Eye disorders
anterior chamber flare † 1	5/28 (17.86%)	5
conjunctival hemorrhage † 1	10/28 (35.71%)	10
eye pain † 1	5/28 (17.86%)	5
1 Term from vocabulary, MedDRA; † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Terry O'Neal, Chief Operating Officer
• Organization: jCyte, Inc
• Phone: 949-243-0688
• EMail: terry.oneal@jcyte.com

• Responsible Party: jCyte, Inc
• ClinicalTrials.gov Identifier: NCT02320812
• Other Study ID Numbers: JC-01
• First Submitted: December 16, 2014
• First Posted: December 19, 2014
• Results First Submitted: August 7, 2018
• Results First Posted: March 5, 2019
• Last Update Posted: March 5, 2019