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Trial record 2 of 2 for:    retinitis pigmentosa jcyte

Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa

This study is currently recruiting participants.
Verified October 2017 by jCyte, Inc
Sponsor:
ClinicalTrials.gov Identifier:
NCT03073733
First Posted: March 8, 2017
Last Update Posted: October 16, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
jCyte, Inc
  Purpose
This study evaluates the changes in visual function at 12 months following a single injection of human retinal progenitor cells compared to sham treated controls in a cohort of adult subjects with RP.

Condition Intervention Phase
Retinitis Pigmentosa Biological: human retinal progenitor cells Other: Mock injection Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Outcomes Assessor)
Masking Description:
Subjects, their family members and clinical staff performing key efficacy assessments will be masked to the randomization assignment of subjects. Due to the nature of some safety assessments and the sham treatment, not all personnel can be masked.
Primary Purpose: Treatment
Official Title: A Prospective, Multicenter, Randomized, Study of the Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)

Resource links provided by NLM:


Further study details as provided by jCyte, Inc:

Primary Outcome Measures:
  • best corrected visual acuity [ Time Frame: 12 months ]
    assessed by E-ETDRS


Secondary Outcome Measures:
  • mobility [ Time Frame: 12 months ]
    maze testing

  • effect of treatment on functional vision [ Time Frame: 12 months ]
    assessed by visual quality of life evaluation

  • visual fields [ Time Frame: 12 months ]
    Goldmann

  • contrast sensitivity [ Time Frame: 12 months ]
    assessment of the ability to distinguish between finer and finer increments of light versus dark (contrast)

  • safety of intravitreal injection of hRPC [ Time Frame: 12 months ]
    assessed by treatment emergent adverse events, immunogenicity and safety visual assessments


Estimated Enrollment: 85
Actual Study Start Date: March 1, 2017
Estimated Study Completion Date: January 2021
Estimated Primary Completion Date: August 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Test (jCell injection) dose level 1
single intravitreal injection of 3.0 x 10e6 human retinal progenitor cells into the eye with the poorest visual acuity or, if vision is comparable in both eyes, the non-dominant eye
Biological: human retinal progenitor cells
live suspension of 3 or 4 x 10e6 human retinal progenitor cells (hRPC) suspended in clinical grade medium injected intravitreally under local anesthesia
Other Name: jCell
Sham treated Control
a mock injection will be performed on the eye with the poorest vision in each Control subject (designated as the "study eye")
Other: Mock injection
pressing the hub of a syringe with no needle against the eye to mimic intravitreal injection
Experimental: test (jCell injection) dose level 2
single intravitreal injection of 4.0 x 10e6 human retinal progenitor cells into the eye with the poorest visual acuity or, if vision is comparable in both eyes, the non-dominant eye
Biological: human retinal progenitor cells
live suspension of 3 or 4 x 10e6 human retinal progenitor cells (hRPC) suspended in clinical grade medium injected intravitreally under local anesthesia
Other Name: jCell

Detailed Description:
There is no effective treatment for RP; once photoreceptors are lost, they do not regenerate. The rate of deterioration of vision varies from person to person, with most people with RP legally blind by age 40. Preclinical studies demonstrated that transplantation of retinal progenitor cells into the eye can result in both photoreceptor replacement and significant slowing of host photoreceptor loss. Thus, the primary goal of this therapy is to preserve, and potentially improve, vision by intervening in the disease at a time when dystrophic host photoreceptors can be protected and reactivated. Based on the demonstration of acceptable safety and tolerability in a phase 1/2a study, this phase 2b study is designed as a controlled comparison of the changes in visual function and functional vision in subjects who receive a single jCell injection in comparison to a comparable sham-treated control group of subjects with RP.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Clinical diagnosis of RP confirmed by ERG and willing to consent to mutation typing, if not already done Best corrected visual acuity (BCVA) 20/80 or worse and no worse than 20/800 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 Treatment with corticosteroids or any investigational or neuroprotectant therapy within 90 days of enrollment Cataract surgery within 3 months prior to enrollment

  Contacts and Locations
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): NCT03073733


Locations
United States, California
Gavin Herbert Eye Inst, Univ Cal Irvine Recruiting
Irvine, California, United States, 92697
Contact: Rosie Magallon    949-824-8297    rmagallo@uci.edu   
Principal Investigator: Baruch Kuppermann, MD         
Retina-Vitreous Associates Medical Group Recruiting
Los Angeles, California, United States, 90074
Contact: Janet Kurokouchi    310-289-2478 ext 123    jkurokouchi@laretina.com   
Principal Investigator: David Boyer, MD         
Sponsors and Collaborators
jCyte, Inc
Investigators
Principal Investigator: Barruch Kuppermann, MD UCI
  More Information

Responsible Party: jCyte, Inc
ClinicalTrials.gov Identifier: NCT03073733     History of Changes
Other Study ID Numbers: JC-02
First Submitted: March 2, 2017
First Posted: March 8, 2017
Last Update Posted: October 16, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Retinitis
Retinitis Pigmentosa
Cone-Rod Dystrophies
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn