SUNIMUD: Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy

Sponsor

Charite University, Berlin, Germany (Other)

Overall Status

Completed

CT.gov ID

NCT01183767

Collaborator

(none)

Enrollment

Locations

Arms

92.2

Actual Duration (Months)

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Duchenne Muscular Dystrophy	Drug: Epigallocatechin-Gallate Drug: Placebo	Phase 2/Phase 3

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 Design

Study Type:

Interventional

Actual Enrollment :

33 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy

Actual Study Start Date :

Dec 30, 2010

Actual Primary Completion Date :

Sep 6, 2018

Actual Study Completion Date :

Sep 6, 2018

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: EGCG Epigallocatechin-Gallate (EGCG)	Drug: Epigallocatechin-Gallate EGCG in a dosage of up to 10mg/kg body weight
Placebo Comparator: Placebo	Drug: Placebo

Arm

Intervention/Treatment

Active Comparator: EGCG

Epigallocatechin-Gallate (EGCG)

Drug: Epigallocatechin-Gallate

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

Placebo Comparator: Placebo

Drug: Placebo

Outcome Measures

Primary Outcome Measures

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

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

5 Years to 10 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	Charité University Berlin	Berlin	Germany	10405
2	DRK Kliniken Berlin, Westend, Pädiatrie	Berlin	Germany	14050
3	Diakonisches Werk Oldenburg SPZ	Oldenburg	Germany