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The purpose of this study is to test the safety and effectiveness of an autologous bone marrow-derived stem/progenitor cells administered intravitreously in the subjects with degenerative diseases of the retina.
Degenerative diseases of the retina are challenging for ophthalmologists. This is a common term that covers heterogenous group of diseases, f.e. retinitis pigmentosa, Stargardt disease, Best's disease or age related macular degeneration. Undetermined etiology, wide range of factors that may trigger the onset of the disease and modulate it's course impede the implementation of an effective treatment. Currently, a stem cells therapy seems to be promising option in patients with degenerative diseases of the retina. The purpose of this prospective, nonrandomized, open label, pilot study is to conduct the investigation of the safety and efficacy of intravitreal injection of autologous bone marrow-isolated stem/progenitor cells with different selected phenotypes into the subjects with degenerative diseases of the retina. Especially, this clinical trial is designated to test the therapeutic (pro-regenerative and neuro-protective) functions of different stem/progenitor cell populations able to secrete bioactive neurotrophic factors. All patients enrolled will have a documented history of degenerative disease of the retina prior to study enrollment. Next, autologous bone marrow-isolated stem/progenitor cells intravitreous injection will be performed. Finally, treatment safety, adverse events and exploratory parameters, including best corrected visual acuity, visual field and electroretinography parameters, to establish disease progression rate will be recorded throughout the duration of the post-treatment follow up period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Stem/progenitor cells transplantation | Active Comparator | Intervention: A single intravitreal injection of autologous bone marrow-derived stem/progenitor cells will be performed. |
|
| Standard treatment of degenerative disease of retina | Sham Comparator | Symptomatic treatment of degenerative disease of retina without biologic cell-based treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Stem/progenitor cells transplantation | Biological | Human autologous bone marrow-derived stem/progenitor cell transplantation administered as an intravitreal injection in patients with degenerative disease of retina. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | Confirm the safety of autologous bone marrow stem/progenitor cell intravitreal injection in enrolled patients by repeated follow-up over one year with clinical evaluations. | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy of autologous bone marrow stem/progenitor intravitreal injection in enrolled patients. | Best corrected visual acuity, ETDRS chart [number of letters] | 12 months |
| Intraocular pressure |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Bogusław Machaliński, MD, PhD | Pomeranian Medical University | Study Chair |
| Anna Machalińska, MD, PhD | Pomeranian Medical University | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| I Department of Ophthalmology | Szczecin | 70-111 | Poland |
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| ID | Term |
|---|---|
| D012162 | Retinal Degeneration |
| D012174 | Retinitis Pigmentosa |
| D008268 | Macular Degeneration |
| D000080362 | Stargardt Disease |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D012164 | Retinal Diseases |
| D058499 | Retinal Dystrophies |
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Pascal tonometer [mmHg]
| 12 months |
| Optic disk retinal nerve fiber layer | optical coherence tomography [um] | 12 months |
| Central macular thickness | optical coherence tomography [um] | 12 months |
| Ganglion cell complex thickness | optical coherence tomography [um] | 12 months |
| Choroidal thickness | Enhanced depth imaging optical coherence tomography [um] | 12 months |
| Choroidal volume | Enhanced depth imaging optical coherence tomography [mm3] | 12 months |
| Computed perimetry (30-2 and 10-2 module) | mean deviation, pattern standard deviation [B] | 12 months |
| Goldmann perimetry with color filters | [degrees] | 12 months |
| Contrast sensitivity | Pelli-Robson chart [number of letters] | 12 months |
| Function of the photoreceptors (rods and cones), inner retinal cells (bipolar and amacrine cells), and ganglion cells. | Electroretinography (ERG) examination:
| 12 months |
| Function of the photoreceptors | Multifocal electroretinography (mfERG) examination:
| 12 months |
| Function of ganglion cells | Pattern electroretinography (PERG) examination:
| 12 months |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |