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

Phase 1

Completed

• Conditions: Retinitis Pigmentosa (RP)
• Interventions: Biological: human retinal progenitor cells

• Registration Number: NCT02320812
• Lead Sponsor: jCyte, Inc

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 Timeline

• Study First Submitted: 2014-12-16
• Study First Submitted QC: 2014-12-16
• Study First Posted: 2014-12-19
• Study Start: 2015-06
• Primary Completion: 2017-07-19
• Study Completion: 2017-07-19
• Results First Submitted: 2018-08-07
• Status Verified: 2019-03
• Results First Submitted QC: 2019-03-04
• Last Update Submitted: 2019-03-04
• Results First Posted: 2019-03-05
• Last Update Posted: 2019-03-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 28

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treated subjects	human retinal progenitor cells	human retinal progenitor cells

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Change in Mean Best Corrected Visual Acuity (BCVA)	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.

Trial Locations

Locations (2)

The Gavin Herbert Eye Institute, Univ California Irvine; 🇺🇸 Irvine, California, United States

Retina-Vitreous Associates Medical Group; 🇺🇸 Los Angeles, California, United States