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)

Sponsors

Lead Sponsor: jCyte, Inc

Collaborator: California Institute for Regenerative Medicine (CIRM)

Source jCyte, Inc
Brief Summary

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.

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.

Overall Status Active, not recruiting
Start Date March 1, 2017
Completion Date January 2021
Primary Completion Date September 2020
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
best corrected visual acuity 12 months
Secondary Outcome
Measure Time Frame
mobility 12 months
effect of treatment on functional vision 12 months
visual fields 12 months
contrast sensitivity 12 months
safety of intravitreal injection of hRPC 12 months
Enrollment 84
Condition
Intervention

Intervention Type: Biological

Intervention Name: human retinal progenitor cells

Description: 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

Intervention Type: Other

Intervention Name: Mock injection

Description: pressing the hub of a syringe with no needle against the eye to mimic intravitreal injection

Arm Group Label: Sham treated Control

Eligibility

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

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Barruch Kuppermann, MD Principal Investigator UCI
Location
Facility:
Gavin Herbert Eye Inst, Univ Cal Irvine | Irvine, California, 92697, United States
Retina-Vitreous Associates Medical Group | Los Angeles, California, 90074, United States
Ophthalmic Consultants of Boston | Boston, Massachusetts, 02114, United States
Location Countries

United States

Verification Date

February 2020

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 3
Arm Group

Label: Test (jCell injection) dose level 1

Type: Experimental

Description: 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

Label: Sham treated Control

Type: Other

Description: a mock injection will be performed on the eye with the poorest vision in each Control subject (designated as the "study eye")

Label: test (jCell injection) dose level 2

Type: Experimental

Description: 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

Patient Data No
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

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.

Source: ClinicalTrials.gov