Back to resultsSafety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis Pigmentosa
Primary Purpose
Retinitis Pigmentosa (RP)
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
human retinal progenitor cells
Sponsored by
About this trial
This is an interventional treatment trial for Retinitis Pigmentosa (RP)
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
Sites / Locations
- The Gavin Herbert Eye Institute, Univ California Irvine
- Retina-Vitreous Associates Medical Group
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Treated subjects
Arm Description
human retinal progenitor cells
Outcomes
Primary Outcome Measures
Number of Subjects With Adverse Events as a Measure of Safety and Tolerability
proportion of subjects with treatment emergent adverse events (TEAE), related TEAE and severe TEAE
Secondary Outcome Measures
Change in Mean Best Corrected Visual Acuity (BCVA)
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.
Full Information
NCT ID
NCT02320812
First Posted
December 16, 2014
Last Updated
March 4, 2019
Sponsor
jCyte, Inc
Collaborators
California Institute for Regenerative Medicine (CIRM)
1. Study Identification
Unique Protocol Identification Number
NCT02320812
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)
Study Type
Interventional
2. Study Status
Record Verification Date
March 2019
Overall Recruitment Status
Completed
Study Start Date
June 2015 (undefined)
Primary Completion Date
July 19, 2017 (Actual)
Study Completion Date
July 19, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
jCyte, Inc
Collaborators
California Institute for Regenerative Medicine (CIRM)
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinitis Pigmentosa (RP)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
28 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Treated subjects
Arm Type
Experimental
Arm Description
human retinal progenitor cells
Intervention Type
Biological
Intervention Name(s)
human retinal progenitor cells
Other Intervention Name(s)
jCell
Intervention Description
single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
Primary Outcome Measure Information:
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
Secondary Outcome Measure Information:
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Baruch Kuppermann, MD
Organizational Affiliation
Gavin Herbert Eye Institute, UCI, Irvine, CA
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Gavin Herbert Eye Institute, Univ California Irvine
City
Irvine
State/Province
California
Country
United States
Facility Name
Retina-Vitreous Associates Medical Group
City
Los Angeles
State/Province
California
ZIP/Postal Code
90074
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis Pigmentosa
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