Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis Pigmentosa
A Prospective, Multicenter, Open-Label, Single-Arm Study of the Safety and Tolerability of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)
  • Phase

    Phase 1/Phase 2
  • Study Type

  • Study Participants

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.
The primary purpose of this study is to test the safety and tolerability of the administration of a single dose of jCell to adults with retinitis pigmentosa.

The goal of jCell therapy is to preserve vision by intervening in the disease at a time when host photoreceptors can be protected and potentially reactivated.

Human allogeneic retinal progenitor cells will be injected into adults with advanced RP to see if the procedure is safe, if the cells survive, and whether they have any impact on the visual status of the patients.
Study Started
Jun 30
Primary Completion
Jul 19
Study Completion
Jul 19
Results Posted
Mar 05
Last Update
Mar 05

Biological human retinal progenitor cells

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

  • Other names: jCell

Treated subjects Experimental

human retinal progenitor cells


Inclusion Criteria:

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

Exclusion Criteria:

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


Treated Subjects

All Events

Event Type Organ System Event Term Treated Subjects

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

Treated Subjects

Subjects with grade 3 TEAE

Subjects with related TEAE

Subjects with TEAE

Change in Mean Best Corrected Visual Acuity (BCVA)

change in mean best corrected visual acuity in test eye versus untreated eye; BCVA is measured using an eye chart and is reported as the number of letters read correctly (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). A positive change from baseline indicates an improvement and a negative change from baseline indicates a worsening. Change between baseline and month 12 is reported.

0.5 Million Cells Cohort

number of letters read correctly (Mean)
Full Range: -3.0 to 11.0

1.0 Million Cells Cohort

number of letters read correctly (Mean)
Full Range: -3.0 to 11.0

2.0 Million Cells Cohort

number of letters read correctly (Mean)
Full Range: 0.0 to 8.0

3.0 Million Cells Cohort

number of letters read correctly (Mean)
Full Range: 0.0 to 12.0

Age, Continuous

years (Mean)
Standard Deviation: 14.7

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Region of Enrollment

Sex: Female, Male

Overall Study

Treated Subjects