Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the short-term safety of a single intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa.

Detailed Description
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A prospective phase I, nonrandomized open-label study of retinitis pigmentosa patients with best-corrected ETDRS visual acuity (BCVA) worse than 20/200. Standardized ophthalmic evaluation will be perform at baseline and at weeks 1, 4,12 and 24 (±1) following intravitreal injection of 10 x 106 bone marrow stem cells/ 0,1ml . Three measures will be used to evaluate the short-term safety of intravitreal of ABMDSC: 1) severe visual loss, defined as a drop in 15 letters on ETDRS visual acuity scale; 2) decrease in ERG response; 3) decrease in 5 square degrees on visual field; secondary safety outcomes : 1) increase in intra-ocular inflammation defined herein as anterior chamber cells and flare higher than 3+ for more than 1 month after injection according to a classification described elsewhere ; 2) decrease in CMT more than 50um; 3)genesis of abnormal tissues (teratomas) or tumors; 4) qualitative changes in retinal or choroidal perfusion, like macular nonperfusion. Secondary outcome measures will be used to evaluate the short-term efficiency of intravitreal of ABMDSC: 1) improvement in ERG response; 2) increase in visual field: 3) increase in CSMT > 50um and not related to macular edema; 4) increase > 5 letters on BCVA

Official Title
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Phase 1 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa

Conditions
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Retinitis Pigmentosa

Intervention / Treatment
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* Biological: intravitreal injection of autologous bone marrow stem cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: diagnosis of retinitis pigmentosa logarithm of minimum angle of resolution (logMAR) BCVA of 1.0 (Snellen equivalent, 20/200) or worse Exclusion Criteria: previous ocular surgery other than cataract presence of cataract or other media opacity that would influence ocular fundus documentation and adequate ERG and visual field evaluation other ophthalmic disease like glaucoma and uveitis previous history of blood disorders like leukemia known allergy to fluorescein or indocyanine green known coagulation abnormalities or current use of anticoagulative medication other than aspirin.

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
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Test group<br>open-label study of retinitis pigmentosa patients with best-corrected visual acuity (BCVA) worse than 20/200. | Biological: intravitreal injection of autologous bone marrow stem cells<br>* One intravitreal injection of a 0.1-ml cell suspension containing around 10x106 bone marrow mononuclear stem cells(BMMSC). All treatments were performed by a single retinal specialist using topical proparacaine drops under sterile conditions (eyelid speculum and povidone-iodine). Autologous BMMSC were injected into the vitreous cavity using a 27 gauge needle inserted through the inferotemporal pars plana 3.0 - 3.5 mm posterior to the limbus. After the injection, central retinal artery perfusion was confirmed with indirect ophthalmoscopy. Patients were instructed to instill one drop of 0.3% ciprofloxacin into the injected eye four times daily for 1 week after the procedure.<br>* Other names: Autologous bone marrow mononuclear stem cells;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| severe visual loss, defined as a drop in 15 letters on ETDRS visual acuity scale |  | 3-6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Retinitis pigmentosa, stem cell, bone marrow