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

Primary Purpose

Retinitis Pigmentosa

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
human retinal progenitor cells
Sponsored by
jCyte, Inc
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Retinitis Pigmentosa

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Gavin Herbert Eye Inst, Univ Cal Irvine
  • Retina-Vitreous Associates Medical Group
  • Ophthalmic Consultants of Boston

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Retreated subjects

Arm Description

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

Outcomes

Primary Outcome Measures

Safety of Intravitreal Injection of hRPC
Assessed by percentage of subjects with treatment emergent adverse events

Secondary Outcome Measures

Best Corrected Visual Acuity
Assessed by E-ETDRS
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
Contrast Sensitivity
The Beethoven System will be used to capture the peak mean contrast sensitivity threshold value at any given spatial frequency
Mobility
Maze testing

Full Information

First Posted
October 16, 2020
Last Updated
August 31, 2023
Sponsor
jCyte, Inc
Collaborators
California Institute for Regenerative Medicine (CIRM)
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1. Study Identification

Unique Protocol Identification Number
NCT04604899
Brief Title
Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa
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)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
December 1, 2020 (Actual)
Primary Completion Date
March 22, 2022 (Actual)
Study Completion Date
March 22, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
jCyte, Inc
Collaborators
California Institute for Regenerative Medicine (CIRM)

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinitis Pigmentosa

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Retreated subjects
Arm Type
Experimental
Arm Description
Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study.
Intervention Type
Biological
Intervention Name(s)
human retinal progenitor cells
Other Intervention Name(s)
jCell
Intervention Description
single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)
Primary Outcome Measure Information:
Title
Safety of Intravitreal Injection of hRPC
Description
Assessed by percentage of subjects with treatment emergent adverse events
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Best Corrected Visual Acuity
Description
Assessed by E-ETDRS
Time Frame
12 months
Title
Visual Fields
Description
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
Time Frame
12 months
Title
Contrast Sensitivity
Description
The Beethoven System will be used to capture the peak mean contrast sensitivity threshold value at any given spatial frequency
Time Frame
12 months
Title
Mobility
Description
Maze testing
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mitul Mehta, MD
Organizational Affiliation
University of California, Irvine/Gavin Herbert Eye Institute
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
David Liao, MD
Organizational Affiliation
Retina-Vitreous Associates Medical Group, Los Angeles CA
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Anthony Jospeh, MD
Organizational Affiliation
Ophthalmic Consultants of Boston
Official's Role
Principal Investigator
Facility Information:
Facility Name
Gavin Herbert Eye Inst, Univ Cal Irvine
City
Irvine
State/Province
California
ZIP/Postal Code
92697
Country
United States
Facility Name
Retina-Vitreous Associates Medical Group
City
Los Angeles
State/Province
California
ZIP/Postal Code
90074
Country
United States
Facility Name
Ophthalmic Consultants of Boston
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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