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

NCT ID: NCT04604899

Last Updated: 2024-07-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-12-01
• Study Completion Date: 2022-03-22

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.

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.

Conditions

Retinitis Pigmentosa

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Retreated subjects

Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study.

Group Type: EXPERIMENTAL

human retinal progenitor cells

Intervention Type: BIOLOGICAL

single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)

Interventions

human retinal progenitor cells

single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)

Intervention Type: BIOLOGICAL

Other Intervention Names

jCell

Eligibility 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

California Institute for Regenerative Medicine (CIRM)

OTHER

Sponsor Role: collaborator

jCyte, Inc

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Mitul Mehta, MD

Role: PRINCIPAL_INVESTIGATOR

University of California, Irvine/Gavin Herbert Eye Institute

David Liao, MD

Role: PRINCIPAL_INVESTIGATOR

Retina-Vitreous Associates Medical Group, Los Angeles CA

Anthony Jospeh, MD

Role: PRINCIPAL_INVESTIGATOR

Ophthalmic Consultants of Boston

Locations

Gavin Herbert Eye Inst, Univ Cal Irvine

Irvine, California, United States

Retina-Vitreous Associates Medical Group

Los Angeles, California, United States

Ophthalmic Consultants of Boston

Boston, Massachusetts, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

JC02-2

Identifier Type: -

Identifier Source: org_study_id