Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa

• ClinicalTrials.gov Identifier: NCT04604899
• Recruitment Status: Completed
• First Posted: October 27, 2020
• Last Update Posted: August 10, 2022
• Sponsor: jCyte, Inc
• Collaborator: California Institute for Regenerative Medicine (CIRM)
• Information provided by (Responsible Party): jCyte, Inc

Study Description

Brief Summary:

The primary objective of the study is to assess the safety of repeat injection of human retinal progenitor cells (jCell) in adult subjects with RP that have previously been treated with jCell.

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

Detailed Description:

This is a prospective, multi-center, single arm, Phase 2 study of human retinal progenitor cells (jCell) for the treatment of retinitis pigmentosa (RP). The study will include only subjects previously treated with jCell.

To assess reinjection of a previously treated eye, subjects who have previously been treated with jCell and desire a second treatment in the same eye will be enrolled. Subjects must have completed at least 12 months of follow up since the prior injection of jCell. Subjects who have had both eyes previously treated with jCell will only have one eye retreated; the eye to be retreated will preferably be the better seeing eye, but exceptions may be made by the study investigator, taking into consideration BCVA, prior response to treatment, and any other medical conditions that may indicate which eye is the best candidate for retreatment. Subjects will be followed for 12 months for safety and efficacy.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 30 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase 2 Study of the Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)
• Actual Study Start Date: December 1, 2020
• Actual Primary Completion Date: March 22, 2022
• Actual Study Completion Date: March 22, 2022

Arms and Interventions

Arm	Intervention/treatment
Experimental: Retreated subjects; Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study.	Biological: human retinal progenitor cells; single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC); Other Name: jCell

Outcome Measures

Primary Outcome Measures :

1. Safety of intravitreal injection of hRPC [ Time Frame: 12 months ]
   Assessed by proportion of subjects with treatment emergent adverse events

Secondary Outcome Measures :

1. Best corrected visual acuity [ Time Frame: 12 months ]
   Assessed by E-ETDRS
2. Visual fields [ Time Frame: 12 months ]
   The Octopus 900 will be used for kinetic visual field testing using a specified target of V4e for more severe subjects and a target of III4e and V4e for better seeing subjects
3. Contrast sensitivity [ Time Frame: 12 months ]
   The Beethoven System will be used to capture the peak mean contrast sensitivity threshold value at any given spatial frequency
4. Mobility [ Time Frame: 12 months ]
   Maze testing

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:

1. Willing to give written informed consent, able to make the required study visits and follow study protocol instructions.
2. Completed the 12 months of follow up in the subject's most recent jCell study and did not withdraw from the study for any reason.

3. Adequate organ function:

   • blood counts (hematocrit, Hgb, WBC, platelets and differential) within normal range, or if outside of normal range, not clinically significant as judged by the investigator
   • liver function: alanine transaminase [ALT] and aspartate transaminase [AST] ≤2 times the upper limit of the normal range
   • total bilirubin ≤1.5 times the upper limit of the normal range
   • renal function: serum creatinine ≤1.25 times the upper limit of the normal range
4. A female patient of childbearing potential (not surgically sterilized and less than one year postmenopausal) must have a negative pregnancy test (urine human chorionic gonadotropin) at entry (prior to injection) and must have used medically accepted contraception for at least one month prior to treatment. Women of childbearing potential and men must be advised to use a medically accepted method of contraception for at least 12 months following treatment.

Exclusion Criteria:

1. Malignancy, end-stage major organ disease (heart failure, significant arrhythmias, stroke or transient ischemic attacks, diabetes, immunosuppressive or autoimmune state, major psychiatric disorder, epilepsy, thyroid disease, COPD, renal failure, or any chronic systemic disease requiring continuous treatment with systemic steroids, anticoagulants or immunosuppressive agents.
2. History of eye disease other than RP that impairs visual function, including retinal vascular disease, elevated intraocular pressure/glaucoma, severe posterior uveitis, clinically significant macular edema, media opacity precluding visual exam, amblyopia and/or longstanding constant strabismus, as well as patients who require other intravitreal therapies
3. Allergy to penicillin or streptomycin.
4. Adverse reaction to DMSO.
5. Unable or unwilling to undergo pupil dilation, topical anesthesia or any protocol-required procedure.
6. Women who are nursing or who are planning to nurse during the 12 months that would follow study treatment.
7. Any circumstance that in the opinion of the investigator, would interfere with participation in, or compliance with the study protocol
8. Treatment with corticosteroids (systemic, periocular or intravitreal) or any other non-approved, experimental, investigational or neuroprotectant therapy (systemic, topical, intravitreal) in either eye within 90 days of planned second injection.
9. Cataract surgery within three months prior to treatment or anticipated to need cataract surgery within a year of treatment

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: Mitul Mehta, MD; University of California, Irvine/Gavin Herbert Eye Institute
• Principal Investigator: David Liao, MD; Retina-Vitreous Associates Medical Group, Los Angeles CA
• Principal Investigator: Anthony Jospeh, MD; Ophthalmic Consultants of Boston

More Information

• Responsible Party: jCyte, Inc
• ClinicalTrials.gov Identifier: NCT04604899
• Other Study ID Numbers: JC02-2
• First Posted: October 27, 2020
• Last Update Posted: August 10, 2022
• Last Verified: August 2022

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