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

jCyte

Status and phase: Completed; Phase 2; Phase 1

Conditions: Retinitis Pigmentosa (RP)

Treatments: Biological: human retinal progenitor cells

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02320812

JC-01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 28 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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28 participants in 1 patient group

Treated subjects

Experimental group

Description:

human retinal progenitor cells

Treatment:

Biological: human retinal progenitor cells