Phase 1 Study of Intravitreal Autologous CD34+ Stem Cell Therapy for Retinitis Pigmentosa

Study Overview
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Brief Summary
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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.

Official Title
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Phase 1 Study of the Feasibility and Safety of Intravitreal Autologous Bone Marrow CD34+ Stem Cell Therapy for Eyes With Vision Loss From Retinitis Pigmentosa

Conditions
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Retinitis Pigmentosa

Intervention / Treatment
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* Biological: Intravitreal autologous CD34+ cells


Participation Criteria
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Eligibility Criteria
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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: Other concurrent optic nerve or retinal disease in study eye affecting vision History of active retinal or choroidal neovascularization or macular edema in study eye requiring on-going intravitreal or periocular therapy in study eye Active eye or systemic infection 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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Intravitreal autologous CD34+ cells<br>Intravitreal injection of autologous CD34+ cells harvested from bone marrow under GMP conditions | Biological: Intravitreal autologous CD34+ cells<br>* Autologous CD34+ cells harvested from bone marrow injected intravitreal<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety of intravitreal injection of autologous CD34+ cells | Number and severity of Ocular and systemic adverse events | 6 months | 
| Feasibility of intravitreal injection of autologous CD34+ cells | Number of CD34+ cells harvested for intravitreal injection | Baseline | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Best corrected visual acuity | Change in number of letters read in study eye from baseline | 6 months | 
| Best corrected visual acuity | Change in number of letters read in study eye from baseline | 1 month | 
| Visual Field | Change in visual field area in study eye from baseline | 6 months | 
| Visual Field | Change in visual field area in study eye from baseline | 1 month | 
| Electroretinography | Change in a and b wave amplitude in study eye from baseline | 6 months | 
| Electroretinography | Change in a and b wave amplitude in study eye from baseline | 1 month | 
| National Eye Institute Vision Questionnaire | Change in Vision Questionnaire compared to baseline | 6 months | 
| National Eye Institute Vision Questionnaire | Change in Vision Questionnaire compared to baseline | 1 month | 
| Microperimetry | Change in average threshold from baseline | 6 months | 
| Microperimetry | Change percent reduced sensitivity from baseline | 6 months | 
| Microperimetry | Change in average threshold from baseline | 1 month | 
| Microperimetry | Change in percent reduced sensitivity from baseline | 1 month | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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retinitis pigmentosa, stem cell, retinal degeneration, intravitreal