Overview

This trial is active, not recruiting.

Condition duchenne muscular dystrophy
Treatments epigallocatechin-gallate, placebo
Phase phase 2/phase 3
Sponsor Charite University, Berlin, Germany
Start date December 2010
End date September 2018
Trial size 40 participants
Trial identifier NCT01183767, SUNIMUD

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Active Comparator)
Epigallocatechin-Gallate (EGCG)
epigallocatechin-gallate
EGCG in a dosage of up to 10mg/kg body weight
(Placebo Comparator)
placebo

Primary Outcomes

Measure
safety and tolerability
time frame: 12 months

Secondary Outcomes

Measure
efficacy
time frame: 36 months

Eligibility Criteria

Male or female participants from 5 years up to 10 years old.

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

Additional Information

Official title Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy
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.
Trial information was received from ClinicalTrials.gov and was last updated in January 2016.
Information provided to ClinicalTrials.gov by Charite University, Berlin, Germany.