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

Retinitis Pigmentosa (RP) Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02320812
• Other study ID #: JC-01
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: June 2015
• Est. completion date: July 19, 2017

Study information

• Verified date: March 2019
• Source: jCyte, Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 28
• Est. completion date: July 19, 2017
• Est. primary completion date: July 19, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa
• Retinitis Pigmentosa (RP)

Intervention

Biological:
• human retinal progenitor cells
single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)

Locations

Country	Name	City	State
United States	The Gavin Herbert Eye Institute, Univ California Irvine	Irvine	California
United States	Retina-Vitreous Associates Medical Group	Los Angeles	California

Sponsors (2)

Lead Sponsor	Collaborator
jCyte, Inc	California Institute for Regenerative Medicine (CIRM)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	12 months
Secondary	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.	12 months