Clinical Trial: Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin

Study Status: Completed
Recruit Status: Unknown status
Study Type: Interventional

Official Title: Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin

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.


Detailed Summary:
Sponsor: Centre Hospitalier Universitaire de Nice

Current Primary Outcome: 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


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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.


Original Secondary Outcome: Same as current

Information By: Centre Hospitalier Universitaire de Nice

Dates:
Date Received: April 11, 2011
Date Started: June 2011
Date Completion: June 2012
Last Updated: October 13, 2011
Last Verified: October 2011