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

Overview

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 Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: December 2011

Interventions

  • Drug: Oral erythromycin
    • Severe Dowling Meara EBS patients from 6 months to 8 years old

Arms, Groups and Cohorts

  • Experimental: Oral erythromycin
    • Oral erythromycin

Clinical Trial Outcome Measures

Primary Measures

  • number of patients with decrease of blisters’ number of at least 20% after 3 months of treatment by oral erythromycin
    • Time Frame: at 3 months of treatment
    • 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

Secondary Measures

  • Secondary end points are : effect of 3 months of oral erythromycin on – Global tolerance of treatment.
    • Time Frame: at 3 months 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.
  • Secondary end points are : effect of 3 months of oral erythromycin on – Involved area
    • Time Frame: at 3 months of treatment
    • 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.
  • Secondary end points are : effect of 3 months of oral erythromycin on – pruritus,
    • Time Frame: at 3 months of treatment
    • 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.

Participating in This Clinical Trial

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 andor hepatic Insufficiency
  • Patient taking a medicine against indicated or misadvised in association with the erythromycin

Gender Eligibility: All

Minimum Age: 6 Months

Maximum Age: 8 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Centre Hospitalier Universitaire de Nice
  • Provider of Information About this Clinical Study
    • Principal Investigator: Del Cont Delphine, Dr Christine CHIAVERINI – Centre Hospitalier Universitaire de Nice
  • Overall Official(s)
    • Christine Chiaverini, PH, Principal Investigator, Centre Hospitalier Universitaire de Nice

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