Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate)

NCT00951964 | Completed Phase 2

• 1 other identifier: 09-APN-01
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 5

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.

Conditions

Epidermolysis Bullosa Dystrophica

Outcome Measures

Primary Outcomes (1)

• decrease of number of cutaneous bullosa

  after 4 months of treatment

Secondary Outcomes (2)

• efficacity of treatment

  at 4 , 6, 7 10 months after beginnig of treatment and at year 1

• tolerance tio treatment

  at 1, 4, 6, 7, 10 and 12 months after beginnig the treatment

Study Arms (2)

1

OTHER

patients receive the treatment in first and placebo in second part of study

Drug: Polyphenon E before Placebo

2

OTHER

patients receive placebo in first and treatment in second part of study

Drug: placebo before treatment

Interventions

Polyphenon E before Placebo DRUG

patients receive polyphenon E during 4 months, then 2 months of wash-out and finally 4 months of placebo

1

placebo before treatment DRUG

patients receive 4 months of placebo, then 2 months of wash out et finally 4 months of treatment

2

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• known mutation of COL7A1

You may not qualify if:

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

Sponsors & Collaborators

• Centre Hospitalier Universitaire de Nice: lead

Study Sites (5)

Dermatology Department, Bordeaux University Hospital

Bordeaux, France

Location

Dijon University Hospital, Dermatology Department

Dijon, France

Location

Dermatology Department, Necker Enfants Malades, APHP

Paris, France

Location

Dermatology Department, Saint Louis Hospital, APHP

Paris, France

Location

Toulouse University Hospital, Dermatology Department

Toulouse, France

Location

Related Publications (1)

• 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.

  PMID: 27015660 DERIVED

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Study Officials

• Christine Chiaverini, PhD

  dermatology department, Nice University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 3, 2009
• First Posted: August 4, 2009
• Study Start: October 1, 2010
• Primary Completion: July 1, 2013
• Study Completion: July 1, 2013
• Last Updated: May 7, 2014

Record last verified: 2014-05

Locations

FR (5)