Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy

NCT ID: NCT01183767

Last Updated: 2021-07-29

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 33 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-12-30
• Study Completion Date: 2018-09-06

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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

EGCG

Epigallocatechin-Gallate (EGCG)

Group Type: ACTIVE_COMPARATOR

Epigallocatechin-Gallate

Intervention Type: DRUG

EGCG in a dosage of up to 10mg/kg body weight

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Interventions

Epigallocatechin-Gallate

EGCG in a dosage of up to 10mg/kg body weight

Intervention Type: DRUG

Placebo

Intervention Type: DRUG

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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Charite University, Berlin, Germany

OTHER

Sponsor Role: lead

Responsible Party

Friedemann Paul

PI

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Friedemann Paul, Dr.

Role: STUDY_DIRECTOR

Charite University, Berlin, Germany

Locations

Charité University Berlin

Berlin, , Germany

DRK Kliniken Berlin, Westend, Pädiatrie

Berlin, , Germany

Diakonisches Werk Oldenburg SPZ

Oldenburg, , Germany

Countries

Germany

Other Identifiers

SUNIMUD

Identifier Type: -

Identifier Source: org_study_id