Clinical trial NCT00951964: Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate)
Official title: Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Epidermolysis Bullosa Dystrophica
Lead sponsor: Centre Hospitalier Universitaire de Nice
Sponsor class: OTHER
First posted: 2009-08-04
Enrollment: 18
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: Dystrophic epidermolysis bullosa hereditaria are genodermatosis responsible for formation of cutaneous bullous lesion arising spontaneously or after mechanical trauma.

These lesions are due to mutation on gene COL7A1 coding for collagen VII. There is no treatment available. Cares are consisting to dress lesions and to protect the skin.

The investigators have recently observed on patients having residual expression of collagen VII that phenotype severity is modulated by activation degree of dermic metalloproteinase. The investigators have also observed that epigallocatechin-3-gallate (Polyphenon E®) could be regulated this activity.

The primary purpose of this study is to assessing the efficacity of Polyphenon E to decrease the number of cutaneous bullosa after four month of treatment.

The primary outcome measure is the rate of patient presenting a decrease of 20% or more of the number of cutaneous bullosa.

Secondary outcomes are: severity of mucosa impairment, affected cutaneous surface, the average duration of cicatrisation and treatment tolerance.

This study foresees the inclusion of 22 patients older than 2 years old in 5 centers.

When patients are included, they will be randomized and receive the treatment (or placebo) for 4 months.


Eligibility criteria: Inclusion Criteria:

* known mutation of COL7A1

Exclusion Criteria:

* tea drinkers
* patient receiving induction treatment,protease inhibitor treatment
* liver failure

Primary outcomes:
measure: decrease of number of cutaneous bullosa
timeFrame: after 4 months of treatment
Secondary outcomes:
measure: efficacity of treatment
timeFrame: at 4 , 6, 7 10 months after beginnig of treatment and at year 1
measure: tolerance tio treatment
timeFrame: at 1, 4, 6, 7, 10 and 12 months after beginnig the treatment
Other outcomes:
Locations:
city: Bordeaux
country: France
facility: Dermatology Department, Bordeaux University Hospital
city: Dijon
country: France
facility: Dijon University Hospital, Dermatology Department
city: Paris
country: France
facility: Dermatology Department, Necker Enfants Malades, APHP
city: Paris
country: France
facility: Dermatology Department, Saint Louis Hospital, APHP
city: Toulouse
country: France
facility: Toulouse University Hospital, Dermatology Department
Study officials:
affiliation: dermatology department, Nice University Hospital
name: Christine Chiaverini, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Chiaverini C, Roger C, Fontas E, Bourrat E, Bourdon-Lanoy E, Labreze C, Mazereeuw J, Vabres P, Bodemer C, Lacour JP. Oral epigallocatechin-3-gallate for treatment of dystrophic epidermolysis bullosa: a multicentre, randomized, crossover, double-blind, placebo-controlled clinical trial. Orphanet J Rare Dis. 2016 Mar 25;11:31. doi: 10.1186/s13023-016-0411-5.