Stem Cells Therapy in Degenerative Diseases of the Retina
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diagnosed degenerative disease of the retina,
- •age 18-65 years,
- •best corrected visual acuity max. 0,2 (Snellen letter chart),
- •good understanding of the protocol and willingness to consent,
- •signed informed consent.
排除标准
- •concomitant eye disease (glaucoma, etc.)
- •concomitant of other systemic disease or diseases,
- •inflammation (high protein or lymphocytosis in the CSF), active infections.
- •diabetes,
- •cardio-vascular disorders,
- •autoimmune diseases,
- •renal failure,
- •impaired hepatic function,
- •subject unwilling or unable to comply with the requirements of the protocol,
- •patient has been treated previously with any cellular therapy,
研究组 & 干预措施
Stem/progenitor cells transplantation
Intervention: A single intravitreal injection of autologous bone marrow-derived stem/progenitor cells will be performed.
干预措施: Stem/progenitor cells transplantation (Biological)
Standard treatment of degenerative disease of retina
Symptomatic treatment of degenerative disease of retina without biologic cell-based treatment
干预措施: Stem/progenitor cells transplantation (Biological)
结局指标
主要结局
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
时间窗: 12 months
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.
次要结局
- Efficacy of autologous bone marrow stem/progenitor intravitreal injection in enrolled patients.(12 months)
- Intraocular pressure(12 months)
- Optic disk retinal nerve fiber layer(12 months)
- Central macular thickness(12 months)
- Ganglion cell complex thickness(12 months)
- Choroidal thickness(12 months)
- Choroidal volume(12 months)
- Computed perimetry (30-2 and 10-2 module)(12 months)
- Goldmann perimetry with color filters(12 months)
- Contrast sensitivity(12 months)
- Function of the photoreceptors (rods and cones), inner retinal cells (bipolar and amacrine cells), and ganglion cells.(12 months)
- Function of the photoreceptors(12 months)
- Function of ganglion cells(12 months)
研究者
Marta P. Wiącek
MD, PhD candidate, principal investigator
Pomeranian Medical University Szczecin