Clinical trial NCT01340235: Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin
Official title: Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Epidermolysis Bullosa
Lead sponsor: Centre Hospitalier Universitaire de Nice
Sponsor class: OTHER
First posted: 2011-04-22
Enrollment: 8
Sex: ALL
Minimum age (years): 6
Maximum age (years): 8
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin
timeFrame: at 3 months of treatment
Secondary outcomes:
measure: Secondary end points are : effect of 3 months of oral erythromycin on - Global tolerance of treatment.
timeFrame: at 3 months of treatment
measure: Secondary end points are : effect of 3 months of oral erythromycin on - Involved area
timeFrame: at 3 months of treatment
measure: Secondary end points are : effect of 3 months of oral erythromycin on - pruritus,
timeFrame: at 3 months of treatment
Other outcomes:
Locations:
city: Dijon
country: France
facility: CHU de Dijon
city: Montpellier
country: France
facility: Hôpital Saint Eloi
city: Nice
country: France
facility: CHU de Nice - Hôpital de Cimiez
city: Toulouse
country: France
facility: Hôpital Purpan
Study officials:
affiliation: Centre Hospitalier Universitaire de Nice
name: Christine Chiaverini, PH
role: PRINCIPAL_INVESTIGATOR
References: