Treatment of Infantile and Juvenile Patients With Bardet-Biedl-Syndrome With Metformin. Evaluation of a Visual Improvement as a Side Effect of the Pediatric Treatment of Adipositas - a Prospective Pilot Study Without Control
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 主要终点
- Visual acuity
研究概览
简要总结
In this prospective pilot study without control group children and young adults (10-25 years old) diagnosed with Bardet-Biedl syndrome and treated with Metformin for their adipositas will be evaluated for a possible additional effect of Metformin on visual acuity.
详细描述
Within experiments with animals a positive effect of Metformin concerning the photo receptors was shown. Several patients of the university hospital Tübingen with Bardet-Biedl-Syndrome and Metformin therapy due to their adipositas reported a subjective improvement of the visual acuity which was confirmed by opthalmologic tests.
This will be checked in a prospective pilot study without control group including children and young adults (10-25 years old) diagnosed with Bardet-Biedl syndrome and treated with Metformin for their adipositas. Therefore a possible additional effect of Metformin on visual acuity will be evaluated under controlled conditions.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •clinically confirmed Bardet-Biedl-syndrome
- •Visual acuity between 0.05 and 0.8
- •Age >=10 year to <25 years
- •Visual field III4e or V4e with diameter >=5°, if II4e not seen
- •informed consent of patient and/or legal representative
排除标准
- •Hypoglycaemia (<50mg/dl)
- •Therapy with Metformin within the last three months
- •Participation in another clinical trial
- •pregnancy, lactation
- •any contra indication concerning Metformin therapy
- •Renal failure (creatinine clearance < 60ml/min)
- •any acute disorder accompanied by clouding of consciousness
- •acute or chronic disorders possible accompanied by tissue hypoxia
- •Liver insufficiency, alcohol abuse
- •not fluent in German language
研究组 & 干预措施
Metformin Therapy
Metformin therapy once daily for 24 weeks with a dose of 500, 850 or 1000 mg depending on body weight
干预措施: Metformin (Drug)
结局指标
主要结局
Visual acuity
时间窗: Baseline to day 210
Course of visual acuity according to appropriate table
次要结局
- Visual evoked potential (VEP) - latency time(Baseline to day 210)
- Electroretinogram (ERG) 30-Hz-Flicker(Baseline to day 210)
- FST(Baseline to day 210)
- Number of letters read(Baseline to day 210)
- Results of Kinetic perimetry(Baseline to day 210)
- Electroretinogram (ERG) standard flash(Baseline to day 210)
- Results of Static perimetry(Baseline to day 210)
- Electroretinogram (ERG) dim-flash(Baseline to day 210)
- Electroretinogram (ERG) cone-single-flash(Baseline to day 210)
- Visual evoked potential (VEP) - amplitude(Baseline to day 210)
- optical coherence tomography (OCT)(Baseline to day 210)