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

Study Overview
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Brief Summary
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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
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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.

Official Title
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A Phase 2 Study of the Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)

Conditions
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Retinitis Pigmentosa

Intervention / Treatment
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* Biological: human retinal progenitor cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Willing to give written informed consent, able to make the required study visits and follow study protocol instructions. 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. 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 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: 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. 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 Allergy to penicillin or streptomycin. Adverse reaction to DMSO. Unable or unwilling to undergo pupil dilation, topical anesthesia or any protocol-required procedure. Women who are nursing or who are planning to nurse during the 12 months that would follow study treatment. Any circumstance that in the opinion of the investigator, would interfere with participation in, or compliance with the study protocol 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. Cataract surgery within three months prior to treatment or anticipated to need cataract surgery within a year of treatment

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Retreated subjects<br>Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. | Biological: human retinal progenitor cells<br>* single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)<br>* Other names: jCell;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety of Intravitreal Injection of hRPC | Assessed by percentage of subjects with treatment emergent adverse events | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Best Corrected Visual Acuity | Assessed by E-ETDRS | 12 months | 
| Visual Fields | 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 | 12 months | 
| Contrast Sensitivity | The Beethoven System will be used to capture the peak mean contrast sensitivity threshold value at any given spatial frequency | 12 months | 
| Mobility | Maze testing | 12 months |