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

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ClinicalTrials.gov Identifier: NCT03073733
Recruitment Status : Active, not recruiting
First Posted : March 8, 2017
Last Update Posted : June 25, 2018
Sponsor:
Information provided by (Responsible Party):
jCyte, Inc

March 2, 2017
March 8, 2017
June 25, 2018
March 1, 2017
August 2019   (Final data collection date for primary outcome measure)
best corrected visual acuity [ Time Frame: 12 months ]
assessed by E-ETDRS
effect of treatment on ocular function [ Time Frame: 12 months ]
assessed by full field sensitivity threshold
Complete list of historical versions of study NCT03073733 on ClinicalTrials.gov Archive Site
  • 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
  • effect of treatment on ocular function [ Time Frame: 12 months ]
    assessed by visual acuity
  • effect of treatment on ocular function [ Time Frame: 12 months ]
    assessed by mobility testing
  • effect of treatment on functional vision [ Time Frame: 12 months ]
    assessed by visual quality of life evaluation
  • effect of treatment on ocular function [ Time Frame: 12 months ]
    assessed by Goldmann visual field area
  • safety of intravitreal injection of hRPC [ Time Frame: 12 months ]
    assessed by treatment emergent adverse events, immunogenicity and safety visual assessments
Not Provided
Not Provided
 
Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa
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)
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.
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.
Interventional
Phase 2
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
Retinitis Pigmentosa
  • Biological: human retinal progenitor cells
    live suspension of 3.0 or 6.0 x 10e6 human retinal progenitor cells (hRPC) suspended in clinical grade medium injected intravitreally under local anesthesia
    Other Name: jCell
  • 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 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
    Intervention: Biological: human retinal progenitor cells
  • 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")
    Intervention: Other: Mock injection
  • Experimental: test (jCell injection) dose level 2
    single intravitreal injection of 6.0 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
    Intervention: Biological: human retinal progenitor cells
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
84
70
January 2021
August 2019   (Final data collection date for primary outcome measure)

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

Sexes Eligible for Study: All
18 Years and older   (Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT03073733
JC-02
No
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
jCyte, Inc
jCyte, Inc
Not Provided
Principal Investigator: Barruch Kuppermann, MD UCI
jCyte, Inc
June 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP