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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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:
Collaborator:
California Institute for Regenerative Medicine (CIRM)
Information provided by (Responsible Party):
jCyte, Inc

Study Description
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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.

Condition or disease Intervention/treatment Phase
Retinitis Pigmentosa (RP) Biological: human retinal progenitor cells Phase 1 Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 28 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : June 2015
Actual Primary Completion Date : July 19, 2017
Actual Study Completion Date : July 19, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Safety

Arms and Interventions
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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 Name: jCell


Outcome Measures
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Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02320812


Locations
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United States, California
The Gavin Herbert Eye Institute, Univ California Irvine
Irvine, California, United States
Retina-Vitreous Associates Medical Group
Los Angeles, California, United States, 90074
Sponsors and Collaborators
jCyte, Inc
California Institute for Regenerative Medicine (CIRM)
Investigators
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Principal Investigator: Baruch Kuppermann, MD Gavin Herbert Eye Institute, UCI, Irvine, CA
  Study Documents (Full-Text)

Documents provided by jCyte, Inc:
Study Protocol  [PDF] July 7, 2016
Statistical Analysis Plan  [PDF] November 8, 2017

More Information
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Responsible Party: jCyte, Inc
ClinicalTrials.gov Identifier: NCT02320812    
Other Study ID Numbers: JC-01
First Posted: December 19, 2014    Key Record Dates
Results First Posted: March 5, 2019
Last Update Posted: March 5, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
 Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn