NCT02320812

Brief Summary

This study evaluates the safety and potential activity of a single dose of live human retinal progenitor cells (jCell) administered to adults with retinitis pigmentosa. Four different dose levels of cells will be assessed in each of two groups of patients.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
28

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jun 2015

Typical duration for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 16, 2014

Completed
3 days until next milestone

First Posted

Study publicly available on registry

December 19, 2014

Completed
5 months until next milestone

Study Start

First participant enrolled

June 1, 2015

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 19, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 19, 2017

Completed
1.6 years until next milestone

Results Posted

Study results publicly available

March 5, 2019

Completed
Last Updated

March 5, 2019

Status Verified

March 1, 2019

Enrollment Period

2.1 years

First QC Date

December 16, 2014

Results QC Date

August 7, 2018

Last Update Submit

March 4, 2019

Conditions

Retinitis Pigmentosa (RP)

Outcome Measures

Primary Outcomes (1)

  • Number of Subjects With Adverse Events as a Measure of Safety and Tolerability

    proportion of subjects with treatment emergent adverse events (TEAE), related TEAE and severe TEAE

    12 months

Secondary Outcomes (1)

  • Change in Mean Best Corrected Visual Acuity (BCVA)

    12 months

Study Arms (1)

Treated subjects

EXPERIMENTAL

human retinal progenitor cells

Biological: human retinal progenitor cells

Interventions

human retinal progenitor cellsBIOLOGICAL

single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)

Also known as: jCell
Treated subjects

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Clinical diagnosis of RP confirmed by electroretinogram (ERG) and willing to consent to mutation typing, if not already done
  • Best corrected visual acuity (BCVA) 20/63 or worse and no worse than hand motions (HM)
  • Adequate organ function and negative infectious disease screen
  • Female of childbearing potential must have negative pregnancy test and be willing to use medically accepted methods of contraception throughout the study

You may not qualify if:

  • Eye disease other than RP that impairs visual function
  • Pseudo-RP, cancer-associated retinopathies
  • History of malignancy or other end-stage organ disease, or any chronic disease requiring continuous treatment with system steroids, anticoagulants or immunosuppressive agents
  • Known allergy to penicillin or streptomycin

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

The Gavin Herbert Eye Institute, Univ California Irvine

Irvine, California, United States

Location

Retina-Vitreous Associates Medical Group

Los Angeles, California, 90074, United States

Location

Related Publications (1)

  • Yang J, Kuppermann BD, Liao D, Mehta MC, Hsiang C, Menges S, Boyer DS, Klassen H. Retinal progenitor cells (jCell) for retinitis pigmentosa. Front Cell Neurosci. 2025 Aug 25;19:1646156. doi: 10.3389/fncel.2025.1646156. eCollection 2025.

    PMID: 40927136DERIVED

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal DystrophiesRetinal DegenerationRetinal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

Title
Terry O'Neal, Chief Operating Officer
Organization
jCyte, Inc
terry.oneal@jcyte.com
949-243-0688

Study Officials

  • Baruch Kuppermann, MD

    Gavin Herbert Eye Institute, UCI, Irvine, CA

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 16, 2014

First Posted

December 19, 2014

Study Start

June 1, 2015

Primary Completion

July 19, 2017

Study Completion

July 19, 2017

Last Updated

March 5, 2019

Results First Posted

March 5, 2019

Record last verified: 2019-03

Data Sharing

IPD Sharing
Will not share

Locations

US(2)