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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.
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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Test group | Experimental | open-label study of retinitis pigmentosa patients with best-corrected visual acuity (BCVA) worse than 20/200. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| intravitreal injection of autologous bone marrow stem cells | Biological | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| severe visual loss, defined as a drop in 15 letters on ETDRS visual acuity scale | 3-6 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Rubens C Siqueira, MD,PhD | Research Center Rubens Siqueira,Catanduva Medicine School and São Paulo University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CPRS | São Jose Do Rio Preto | São Paulo | 15010-100 | Brazil |
| Type | Date | Date Unknown |
|---|---|---|
| Release | Nov 18, 2013 | |
| Reset | Jan 9, 2014 | |
| Release | Jan 12, 2014 | |
| Reset | Mar 3, 2014 | |
| Release | Jul 18, 2017 | |
| Reset | Feb 1, 2018 | |
| Release | Oct 15, 2018 | |
| Reset | Feb 19, 2019 |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Nov 18, 2013 | Jan 9, 2014 | |||
| Jan 12, 2014 |
| ID | Term |
|---|---|
| D012174 | Retinitis Pigmentosa |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D058499 | Retinal Dystrophies |
| D012162 | Retinal Degeneration |
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|
|
| Mar 3, 2014 |
| Jul 18, 2017 | Feb 1, 2018 |
| Oct 15, 2018 | Feb 19, 2019 |
| D012164 |
| Retinal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |