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

Retinitis Pigmentosa Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04604899
• Other study ID #: JC02-2
• Status: Completed
• Phase: Phase 2
• Start date: December 1, 2020
• Est. completion date: March 22, 2022

Study information

• Verified date: August 2023
• Source: jCyte, Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: March 22, 2022
• Est. primary completion date: March 22, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa

Intervention

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

Locations

Country	Name	City	State
United States	Ophthalmic Consultants of Boston	Boston	Massachusetts
United States	Gavin Herbert Eye Inst, Univ Cal Irvine	Irvine	California
United States	Retina-Vitreous Associates Medical Group	Los Angeles	California

Sponsors (2)

Lead Sponsor	Collaborator
jCyte, Inc	California Institute for Regenerative Medicine (CIRM)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety of Intravitreal Injection of hRPC	Assessed by percentage of subjects with treatment emergent adverse events	12 months
Secondary	Best Corrected Visual Acuity	Assessed by E-ETDRS	12 months
Secondary	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
Secondary	Contrast Sensitivity	The Beethoven System will be used to capture the peak mean contrast sensitivity threshold value at any given spatial frequency	12 months
Secondary	Mobility	Maze testing	12 months