Retinitis Pigmentosa (RP)
Conditions
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.
Detailed description
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.
Interventions
BIOLOGICALhuman retinal progenitor cells
single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC)
Sponsors
California Institute for Regenerative Medicine (CIRM)
jCyte, Inc
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Subjects With Adverse Events as a Measure of Safety and Tolerability | 12 months | proportion of subjects with treatment emergent adverse events (TEAE), related TEAE and severe TEAE |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Mean Best Corrected Visual Acuity (BCVA) | 12 months | 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Treated Subjects human retinal progenitor cells
human retinal progenitor cells: single intravitreal injection of 0.5 - 3.0 million human retinal progenitor cells (hRPC) | 28 |
| Total | 28 |
Baseline characteristics
| Characteristic | Treated Subjects |
|---|---|
| Age, Continuous | 49.2 years STANDARD_DEVIATION 14.7 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 7 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 21 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 4 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 24 Participants |
| Region of Enrollment United States | 28 participants |
| Sex: Female, Male Female | 17 Participants |
| Sex: Female, Male Male | 11 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 28 |
| other Total, other adverse events | 20 / 28 |
| serious Total, serious adverse events | 1 / 28 |
Outcome results
Primary
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
Time frame: 12 months
Population: all treated subjects
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Treated Subjects | Number of Subjects With Adverse Events as a Measure of Safety and Tolerability | Subjects with TEAE | 25 Participants |
| Treated Subjects | Number of Subjects With Adverse Events as a Measure of Safety and Tolerability | Subjects with related TEAE | 21 Participants |
| Treated Subjects | Number of Subjects With Adverse Events as a Measure of Safety and Tolerability | Subjects with grade 3 TEAE | 1 Participants |
Secondary
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.
Time frame: 12 months
Population: all treated subjects
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Treated Subjects | Change in Mean Best Corrected Visual Acuity (BCVA) | 1.4 number of letters read correctly |
| 1.0 Million Cells Cohort | Change in Mean Best Corrected Visual Acuity (BCVA) | 1.0 number of letters read correctly |
| 2.0 Million Cells Cohort | Change in Mean Best Corrected Visual Acuity (BCVA) | 4.8 number of letters read correctly |
| 3.0 Million Cells Cohort | Change in Mean Best Corrected Visual Acuity (BCVA) | 9.0 number of letters read correctly |