Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy

Phase 2

Completed

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Drug: Placebo; Drug: Epigallocatechin-Gallate

• Registration Number: NCT01183767
• Lead Sponsor: Charite University, Berlin, Germany

Brief Summary

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

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.

Study Timeline

• Study First Submitted: 2010-08-17
• Study First Submitted QC: 2010-08-17
• Study First Posted: 2010-08-18
• Study Start: 2010-12-30
• Primary Completion: 2018-09-06
• Study Completion: 2018-09-06
• Status Verified: 2021-07
• Last Update Submitted: 2021-07-28
• Last Update Posted: 2021-07-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 33

Inclusion Criteria

• Duchenne muscular dystrophy
• age 5-10 years
• ability to walk without support
• informed consent by the parents

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Exclusion Criteria

• another serious organic disease
• further primary psychiatric or neurological diseases
• long-term intake of liver-toxic medicines

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	-
EGCG	Epigallocatechin-Gallate	Epigallocatechin-Gallate (EGCG)

Primary Outcome Measures

Name	Time	Method
safety and tolerability	12 months	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.

Secondary Outcome Measures

Name	Time	Method
efficacy	36 months	changes in the means of the 6 minute walk test (baseline to visit after month 36).

Trial Locations

Locations (3)

Charité University Berlin; 🇩🇪 Berlin, Germany

DRK Kliniken Berlin, Westend, Pädiatrie; 🇩🇪 Berlin, Germany

Diakonisches Werk Oldenburg SPZ; 🇩🇪 Oldenburg, Germany