Clinical trial NCT01183767: Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy
Official title: Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne Muscular Dystrophy
Epigallocatechin-Gallate
Green tea extract
Lead sponsor: Charite University, Berlin, Germany
Sponsor class: OTHER
First posted: 2010-08-18
Enrollment: 33
Sex: ALL
Minimum age (years): 5
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: safety and tolerability
timeFrame: 12 months
Secondary outcomes:
measure: efficacy
timeFrame: 36 months
Other outcomes:
Locations:
city: Berlin
country: Germany
facility: Charité University Berlin
city: Berlin
country: Germany
facility: DRK Kliniken Berlin, Westend, Pädiatrie
city: Oldenburg
country: Germany
facility: Diakonisches Werk Oldenburg SPZ
Study officials:
affiliation: Charite University, Berlin, Germany
name: Friedemann Paul, Dr.
role: STUDY_DIRECTOR
References: