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

Tracking Information

• First Submitted Date: December 16, 2014
• First Posted Date: December 19, 2014
• Results First Submitted Date: August 7, 2018
• Results First Posted Date: March 5, 2019
• Last Update Posted Date: March 5, 2019
• Study Start Date: June 2015
• Actual Primary Completion Date: July 19, 2017 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 4, 2019)

Number of Subjects With Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 12 months ]

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

Original Primary Outcome Measures (submitted: December 16, 2014)

Number of Subjects With Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 12 months ]

adverse events, with emphasis on ophthalmic adverse events

Current Secondary Outcome Measures (submitted: March 4, 2019)

Change in Mean Best Corrected Visual Acuity (BCVA) [ Time Frame: 12 months ]

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.

Original Secondary Outcome Measures (submitted: December 16, 2014)

Effect of treatment on ocular function [ Time Frame: 12 months ]

Best corrected visual acuity, Optical Coherence Tomography, Electroretinogram, Visual Field Testing, Fluorescein Angiography

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis Pigmentosa
• Official Title: A Prospective, Multicenter, Open-Label, Single-Arm Study of the Safety and Tolerability of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)
• Brief Summary: This study evaluates the safety and potential activity of a single dose of live human retinal progenitor cells (jCell) administered to adults with retinitis pigmentosa. Four different dose levels of cells will be assessed in each of two groups of patients.

Detailed Description

The primary purpose of this study is to test the safety and tolerability of the administration of a single dose of jCell to adults with retinitis pigmentosa.

The goal of jCell therapy is to preserve vision by intervening in the disease at a time when host photoreceptors can be protected and potentially reactivated.

Human allogeneic retinal progenitor cells will be injected into adults with advanced RP to see if the procedure is safe, if the cells survive, and whether they have any impact on the visual status of the patients.

• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• 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

single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)

Other Name: jCell

Study Arms

Experimental: Treated subjects

human retinal progenitor cells

Intervention: Biological: human retinal progenitor cells

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 10, 2016): 28
• Original Estimated Enrollment (submitted: December 16, 2014): 16
• Actual Study Completion Date: July 19, 2017
• Actual Primary Completion Date: July 19, 2017 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Clinical diagnosis of RP confirmed by electroretinogram (ERG) and willing to consent to mutation typing, if not already done
• Best corrected visual acuity (BCVA) 20/63 or worse and no worse than hand motions (HM)
• 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02320812
• Other Study ID Numbers: JC-01
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: jCyte, Inc
• Study Sponsor: jCyte, Inc
• Collaborators: California Institute for Regenerative Medicine (CIRM)

Investigators

• Principal Investigator: Baruch Kuppermann, MD; Gavin Herbert Eye Institute, UCI, Irvine, CA

• PRS Account: jCyte, Inc
• Verification Date: March 2019