Autologous Bone Marrow-Derived CD34+, CD133+, and CD271+ Stem Cell Transplantation for Retinitis Pigmentosa

Study Overview
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Brief Summary
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A single arm, single center trial to evaluate the safety and efficacy of autologous purified populations of bone-marrow derived stem cells in patients with Retinitis Pigmentosa (BM-SCs) through a 48 month follow up period.

Detailed Description
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Retinitis pigmentosa (RP) is an inherited disorder of the photoreceptor cells in the retina. Patients may lose vision since they were young or later in life. Currently, there are more than 60 genes identified as the cause of this condition, one of which, RPE65, has been studied in several gene therapy trials for Leber congenital amaurosis with promising results. Another treatment approach for RP is stem cell therapy. Studies in animal models of RP have shown that subretinal injection of bone marrow-derived mesenchymal stem cells may delay degenerative changes of photoreceptor cells. This is a single arm, single center trial to assess the safety and efficacy of purified adult autologous bone marrow derived CD34+, CD133+, and CD271+ stem cells through a 48 month follow-up period. The combination of these three cell types was based on their diverse potentialities to differentiate into specific functional cell types to regenerate damaged retinal tissue, and the availability of clinical-grade purification system (CliniMACS) and microbeads to purify the target cell populations in clinically-approved methods.

Conditions
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Retinitis Pigmentosa

Intervention / Treatment
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* Biological: Stem Cell Transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Retinitis pigmentosa patients diagnosed by ophthalmologists Visual acuity (measured with ETDRS) less than or equal to 20/70 and visual field below 30° central in both eyes. Best corrected visual acuity less than 6/120 by Snellen visual acuity chart Exclusion Criteria: Other eye conditions that could mask the interpretation of the results Unable to return for follow up Underlying diseases including asthma, heart failure, myocardial infarction, liver failure, renal failure Pregnant and lactating women

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 55 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: Stem Cells<br>Intravitreal Injection of bone marrow derived CD34+, CD133+, CD271+ stem cells. | Biological: Stem Cell Transplantation<br>* Bone marrow-derived CD34+, CD133+, CD271+ stem cells in 1.0 ml normal saline will be injected into the vitreous cavity.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| ETDRS Visual acuity change |  | 12 months from baseline | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Quality of Life: Questionnaire VFQ-25 (Visual Function Questionnaire-25) |  | 12 months from baseline | 
| Color Vision: Ishihara Color Test |  | 12 months from baseline | 
| Contrast sensitivity: CSV-1000E |  | 12 months from baseline |