Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy

Study Overview
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Brief Summary
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The aim of this multicentre, prospective, double blind, placebo controlled, randomized pilot study is to investigate safety and tolerance of Epigallocatechin-Gallate (EGCG, the major polyphenol in green tea) in patients with muscular dystrophy of the Duchenne type. In a second step the investigators want to investigate the effect of EGCG on the course of the Duchenne condition.

Detailed Description
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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.

Official Title
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Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Drug: Epigallocatechin-Gallate
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 10 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: EGCG<br>Epigallocatechin-Gallate (EGCG) | Drug: Epigallocatechin-Gallate<br>* EGCG in a dosage of up to 10mg/kg body weight<br>|
| Placebo Comparator: Placebo<br> | Drug: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| safety and tolerability | safety and tolerability in terms of number of adverse events in which a causal relationship with the test substance cannot be excluded, and GLDH values. | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| efficacy | changes in the means of the 6 minute walk test (baseline to visit after month 36). | 36 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Duchenne Muscular Dystrophy, Epigallocatechin-Gallate, Green tea extract