Intravitreal Injection of MSCs in Retinitis Pigmentosa

Study Overview
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Brief Summary
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The purpose of this study is to determine the feasibility and safety of adult human bone marrow-derived mesenchymal stem cells by intravitreal injection in patients with retinitis pigmentosa.

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.

Official Title
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Feasibility and Safety of Adult Human Bone Marrow-Derived Mesenchymal Stem Cells by Intravitreal Injection in Patients With Retinitis Pigmentosa

Conditions
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Retinitis Pigmentosa

Intervention / Treatment
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* Other: BM-MSC


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Retinitis pigmentosa patients diagnosed by ophthalmologists Age 18-65 years old Central visual field less than or equal to 20 degrees Best corrected visual acuity less than 6/120 by Snellen visual acuity chart Electroretinogram nonrecordable or the amplitudes were less than 25% of normal 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: 65 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: Other
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BM-MSC<br>Bone marrow-derived mesenchymal stem cells 1 million cells in balanced salt solution 100 microlitres will be injected into the vitreous cavity. | Other: BM-MSC<br>* Bone marrow-derived mesenchymal stem cells 1 million cells in balanced salt solution 100 microlitres 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 |
| --- | --- | --- |
| Change from baseline in laser flare and cell measurements |  | up to 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in visual function tests |  | up to 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Retinitis pigmentosa, Bone marrow-derived mesenchymal stem cells, Intravitreal injection