Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin
Information source: Centre Hospitalier Universitaire de Nice
Information obtained from ClinicalTrials.gov on February 07, 2013
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Epidermolysis Bullosa
Intervention: Oral erythromycin (Drug)
Phase: Phase 3
Sponsored by: Centre Hospitalier Universitaire de Nice
Official(s) and/or principal investigator(s):
Christine Chiaverini, PH, Principal Investigator, Affiliation: CHU de NIce
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.
Official title: Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin
Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin
Secondary end points are : effect of 3 months of oral erythromycin on - Global tolerance of treatment.
Secondary end points are : effect of 3 months of oral erythromycin on - Involved area
Secondary end points are : effect of 3 months of oral erythromycin on - pruritus,
Minimum age: 6 Months.
Maximum age: 8 Years.
- 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
- 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
Locations and Contacts
CHU de Dijon, Dijon, France; Recruiting
Pierre Vabres, PU-PH, Email: firstname.lastname@example.org
Hôpital Saint Eloi, Montpellier, France; Not yet recruiting
Didier Bessis, PH
CHU de Nice - Hôpital de Cimiez, Nice 06000, France; Recruiting
Christine Chiaverini, PH, Phone: 0033 4 92 03 61 07, Email: email@example.com
Vanina Oliveri, ARC, Phone: 0033 4 92 03 42 54, Email: firstname.lastname@example.org
Hôpital Purpan, Toulouse, France; Recruiting
Juliette MAZEREEUW, PU-PH, Phone: 00 33 5 67 77 81 41, Email: email@example.com
Starting date: June 2011
Last updated: October 13, 2011