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

• ClinicalTrials.gov Identifier: NCT03073733
• Recruitment Status: Active, not recruiting
• First Posted: March 8, 2017
• Last Update Posted: March 5, 2019
• Sponsor: jCyte, Inc
• Collaborator: California Institute for Regenerative Medicine (CIRM)
• Information provided by (Responsible Party): jCyte, Inc

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Retinitis Pigmentosa	Biological: human retinal progenitor cells; Other: Mock injection	Phase 2

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 84 participants
• 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)
• Actual Study Start Date: March 1, 2017
• Estimated Primary Completion Date: August 2019
• Estimated Study Completion Date: January 2021

Arms and Interventions

Arm	Intervention/treatment
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 3.0 or 6.0 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 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	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

Outcome Measures

Primary Outcome Measures :

1. best corrected visual acuity [ Time Frame: 12 months ]
   assessed by E-ETDRS

Secondary Outcome Measures :

1. mobility [ Time Frame: 12 months ]
   maze testing
2. effect of treatment on functional vision [ Time Frame: 12 months ]
   assessed by visual quality of life evaluation
3. visual fields [ Time Frame: 12 months ]
   Goldmann
4. contrast sensitivity [ Time Frame: 12 months ]
   assessment of the ability to distinguish between finer and finer increments of light versus dark (contrast)
5. safety of intravitreal injection of hRPC [ Time Frame: 12 months ]
   assessed by treatment emergent adverse events, immunogenicity and safety visual assessments

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• 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

Locations

United States, California

Gavin Herbert Eye Inst, Univ Cal Irvine

Irvine, California, United States, 92697

Retina-Vitreous Associates Medical Group

Los Angeles, California, United States, 90074

United States, Massachusetts

Ophthalmic Consultants of Boston

Boston, Massachusetts, United States, 02114

Sponsors and Collaborators

jCyte, Inc

California Institute for Regenerative Medicine (CIRM)

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 Posted: March 8, 2017
• Last Update Posted: March 5, 2019
• Last Verified: March 2019

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; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Retinal Dystrophies; Retinal Degeneration; Genetic Diseases, Inborn