Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy
关键词
抽象
描述
Duchenne muscular dystrophy (DMD) is the most frequent neuromuscular condition to occur in childhood and youth. The course of the disease is progressive, and life expectancy is severely curtailed by the participation of the respiratory muscles and/or by progressive cardiomyopathy.
DMD derives from mutations in the DMD gene which leads to a loss of the protein dystrophin. Secondary inflammatory/immunological reactions contribute to the progressive course of the disease (1,2).
No curative therapy yet exists. Administration of steroids is the only established medical treatment. Symptomatic measures are also available, such as orthopaedic operations, the treatment of cardiomyopathy or, in advanced stages, home mechanical ventilation.
In studies involving experiments on cells and animals, Epigallocatechin-Gallate (EGCG, the major polyphenol in green tea) has shown a neuroprotective effect. The neuroprotective mechanism of action is probably based on several factors, including EGCG's modulation of several signal transduction pathways, its influence on the expression of genes regulating cell survival or programmed cell death, as well as its modulation of mitochondrial function.
The mdx mouse is the best-investigated animal model of a dystrophin-negative muscular dystrophy. Administration of EGCG in the mdx mouse led to both a reduction in the proportion of fibre necroses as well as to a less pronounced proliferation of connective tissue in the muscle (3,4), and also to an improvement in clinical symptoms (5,6).
Therefore, the investigators want to investigate safety and tolerance of EGCG in a dosage of up to 10mg/kg in patients with muscular dystrophy of the Duchenne type in this multicentre, prospective, double blind, placebo controlled, randomized pilot study.
日期
最后验证: | 03/31/2020 |
首次提交: | 08/16/2010 |
提交的预估入学人数: | 08/16/2010 |
首次发布: | 08/17/2010 |
上次提交的更新: | 04/13/2020 |
最近更新发布: | 04/14/2020 |
实际学习开始日期: | 12/29/2010 |
预计主要完成日期: | 09/05/2018 |
预计完成日期: | 09/05/2018 |
状况或疾病
干预/治疗
Drug: EGCG
Drug: Placebo
相
手臂组
臂 | 干预/治疗 |
---|---|
Active Comparator: EGCG Epigallocatechin-Gallate (EGCG) | Drug: EGCG EGCG in a dosage of up to 10mg/kg body weight |
Placebo Comparator: Placebo | Drug: Placebo |
资格标准
有资格学习的年龄 | 5 Years 至 5 Years |
有资格学习的性别 | All |
接受健康志愿者 | 是 |
标准 | Inclusion Criteria: - Duchenne muscular dystrophy - age 5-10 years - ability to walk without support - informed consent by the parents Exclusion Criteria: - another serious organic disease - further primary psychiatric or neurological diseases - long-term intake of liver-toxic medicines |
结果
主要结果指标
1. safety and tolerability [12 months]
次要成果指标
1. efficacy [36 months]