Retinitis Pigmentosa
Conditions
Keywords
retinitis pigmentosa, stem cell, retinal degeneration, intravitreal
Brief summary
In this Phase 1 open-labeled prospective study, one eye of each participant with vision loss from retinitis pigmentosa will be administered intravitreal injection of autologous CD34+ stem cells harvested from bone marrow. Each participant will be examined serially for 6 months after study injection to determine safety and feasibility of this intervention.
Interventions
BIOLOGICALIntravitreal autologous CD34+ cells
Autologous CD34+ cells harvested from bone marrow injected intravitreal
Sponsors
Cures Within Reach
The Retina Society
University of California, Davis
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
1\. Age \>18 years of age 2. Visual Acuity at Presentation of 20/100 to Counting Fingers or constriction of peripheral visual field 360 degrees to \<10 degrees by perimetry if BCVA is better than 20/100, duration of vision loss \> 3 months 3. Vision loss due to retinitis pigmentosa 4. The study eye has the worse visual acuity or peripheral constriction. 14. Able and willing to sign consent 15. Able to keep follow-up appointments for at least 6 months
Exclusion criteria
1. Other concurrent optic nerve or retinal disease in study eye affecting vision 2. History of active retinal or choroidal neovascularization or macular edema in study eye requiring on-going intravitreal or periocular therapy in study eye 3. Active eye or systemic infection 4. Significant media opacity in the study eye precluding view of the fundus for examination, photography or optical coherence tomography (OCT) 9\. Other cause for vision loss in the study eye 10. History of coagulopathy or other hematologic abnormality 11. Use of coumadin within 3 days of enrollment 12. Concurrent immunosuppressive therapy 13. History of allergy to fluorescein dye
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety of intravitreal injection of autologous CD34+ cells | 6 months | Number and severity of Ocular and systemic adverse events |
| Feasibility of intravitreal injection of autologous CD34+ cells | Baseline | Number of CD34+ cells harvested for intravitreal injection |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Electroretinography | 6 months | Change in a and b wave amplitude in study eye from baseline |
| Best corrected visual acuity | 6 months | Change in number of letters read in study eye from baseline |
| Microperimetry | 6 months | Change in average threshold from baseline |
| National Eye Institute Vision Questionnaire | 6 months | Change in Vision Questionnaire compared to baseline |
| Visual Field | 6 months | Change in visual field area in study eye from baseline |
Countries
United States
Outcome results
None listed