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

March 4, 2019 updated by: jCyte, Inc

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)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Enrollment (Actual)

28

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Irvine, California, United States
        • The Gavin Herbert Eye Institute, Univ California Irvine
      • Los Angeles, California, United States, 90074
        • Retina-Vitreous Associates Medical Group

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Treated subjects
human retinal progenitor cells
Biological: human retinal progenitor cells
single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
Other Names:
  • jCell

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

jCyte, Inc

Collaborators

California Institute for Regenerative Medicine (CIRM)

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2015

Primary Completion (Actual)

July 19, 2017

Study Completion (Actual)

July 19, 2017

Study Registration Dates

First Submitted

December 16, 2014

First Submitted That Met QC Criteria

December 16, 2014

First Posted (Estimate)

December 19, 2014

Study Record Updates

Last Update Posted (Actual)

March 5, 2019

Last Update Submitted That Met QC Criteria

March 4, 2019

Last Verified

March 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • JC-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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