Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin

Dowling Meara type of epidermolysis bullosa simplex (EBS-DM) is a rare genodermatosis due to keratin 5 and 14 mutation, characterized by skin fragility and spontaneous or post traumatic blisters. Neonatal period and infancy are critical since this autonomic dominant affection usually improves with age. Cyclins seem to be efficient in some cases of EBS but are prohibited in children younger than 8 years old. Erythromycin can be a good alternative in this population due to its antibacterial and anti-inflammatory potential.

The aim of this study is the evaluation of the efficiency of oral erythromycin to decrease the number of cutaneous blisters in severe EBS-DM patients from 6 months to 8 years old after 3 months of treatment.

Primary end point is the number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin.

It is a preliminary study on 8 patients. Treatment is oral erythromycin twice a day during 3 months. Follow up for each patient is 5 months. The duration of the study is 1 year.

Study Overview

• Status: Unknown
• Conditions: Epidermolysis Bullosa
• Intervention / Treatment: Drug: Oral erythromycin

• Study Type: Interventional
• Enrollment (Anticipated): 8
• Phase: Phase 3

Contacts and Locations

Study Locations

• France
  • Dijon, France
    • Recruiting
    • CHU de Dijon
    • Contact: Pierre Vabres, PU-PH; Email: pierre.vabres@chu-dijon.fr
  • Montpellier, France
    • Not yet recruiting
    • Hopital Saint Eloi
    • Contact: Didier Bessis, PH
  • Nice, France, 06000
    • Recruiting
    • CHU de Nice - Hôpital de Cimiez
    • Contact: Vanina Oliveri, ARC; Phone Number: 0033 4 92 03 42 54; Email: oliveri.v@chu-nice.fr
    • Contact: Christine Chiaverini, PH; Phone Number: 0033 4 92 03 61 07; Email: chiaverini.c@chu-nice.fr
  • Toulouse, France
    • Recruiting
    • Hopital Purpan
    • Contact: Juliette MAZEREEUW, PU-PH; Phone Number: 00 33 5 67 77 81 41; Email: mazereeuw-hautier.j@chu-toulouse.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 8 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Severe Dowling Meara EBS patients (2 or more new blisters a day)
• signature of informed consent
• Patient of 2 sexes
• Age from 6 months to 8 years. From this age we consider that the patient will less need this treatment or can take cyclines.
• Systematic Obtaining of the consent lit(enlightened) by the relatives(parents) of the child, after information about the objectives and the constraints of the study.
• Agreement of the minor
• Patient member to the Social Security

Exclusion Criteria:

• Patient allergic to the erythromycin
• Patient presenting an intolerance to the fructose, a syndrome of malabsorption some glucose and some galactose or a deficit sucrase-isomaltase
• Renal and\or hepatic Insufficiency
• Patient taking a medicine against indicated or misadvised in association with the erythromycin

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Oral erythromycin	Drug: Oral erythromycin; Severe Dowling Meara EBS patients from 6 months to 8 years old

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin	Principal end point is evaluated at inclusion and after one month of treatment, 3 months of treatment and 2 months after the end of the treatment	at 3 months of treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Secondary end points are : effect of 3 months of oral erythromycin on - Global tolerance of treatment.	For each patient and globally, the nature, the frequency and the severity of the various unwanted effects will be described on the duration of the study.	at 3 months of treatment
Secondary end points are : effect of 3 months of oral erythromycin on - Involved area	These criteria will be analyzed in comparison with the values to the inclusion (M0). We shall try to estimate the obstinacy of an effect 2 months after the end of the treatment.	at 3 months of treatment
Secondary end points are : effect of 3 months of oral erythromycin on - pruritus,	These criteria will be analyzed in comparison with the values to the inclusion (M0). We shall try to estimate the obstinacy of an effect 2 months after the end of the treatment.	at 3 months of treatment

Collaborators and Investigators

• Sponsor: Centre Hospitalier Universitaire de Nice
• Investigators: Principal Investigator: Christine Chiaverini, PH, Centre Hospitalier Universitaire de Nice

Study record dates

Study Major Dates

• Study Start: June 1, 2011
• Primary Completion (ANTICIPATED): December 1, 2011
• Study Completion (ANTICIPATED): June 1, 2012

Study Registration Dates

• First Submitted: April 11, 2011
• First Submitted That Met QC Criteria: April 21, 2011
• First Posted (ESTIMATE): April 22, 2011

Study Record Updates

• Last Update Posted (ESTIMATE): October 14, 2011
• Last Update Submitted That Met QC Criteria: October 13, 2011
• Last Verified: October 1, 2011

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Epidermolysis Bullosa; Epidermolysis Bullosa Simplex; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Enzyme Inhibitors; Gastrointestinal Agents; Anti-Bacterial Agents; Protein Synthesis Inhibitors; Erythromycin; Erythromycin Estolate; Erythromycin Ethylsuccinate; Erythromycin stearate
• Other Study ID Numbers: 10-PP-19