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

Centre Hospitalier Universitaire de Nice

Status and phase

Unknown

Phase 3

Conditions

Epidermolysis Bullosa

Treatments

Drug: Oral erythromycin

Study type

Interventional

Funder types

Other

Identifiers

NCT01340235

10-PP-19

Details and patient eligibility

About

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.

Enrollment

8 estimated patients

Sex

All

Ages

6 months to 8 years old

Volunteers

No Healthy Volunteers

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