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
-
-
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
|
single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
Other Names:
|
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
Collaborators
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.