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

Epidermolysis Bullosa Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01340235
• Other study ID #: 10-PP-19
• Status: Recruiting
• Phase: Phase 3
• First received: April 11, 2011
• Last updated: October 13, 2011
• Start date: June 2011
• Est. completion date: June 2012

Study information

• Verified date: October 2011
• Source: Centre Hospitalier Universitaire de Nice
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)France: Committee for the Protection of Personnes
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 8
• Est. completion date: June 2012
• Est. primary completion date: December 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Months to 8 Years

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Epidermolysis Bullosa
• Epidermolysis Bullosa Simplex

Intervention

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

Locations

Country	Name	City	State
France	CHU de Dijon	Dijon
France	Hôpital Saint Eloi	Montpellier
France	CHU de Nice - Hôpital de Cimiez	Nice
France	Hôpital Purpan	Toulouse

Sponsors (1)

Lead Sponsor	Collaborator
Centre Hospitalier Universitaire de Nice

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin	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	at 3 months of treatment	Yes
Secondary	Secondary end points are : effect of 3 months of oral erythromycin on - Global tolerance 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.	at 3 months of treatment	Yes
Secondary	Secondary end points are : effect of 3 months of oral erythromycin on - Involved area	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.	at 3 months of treatment	Yes
Secondary	Secondary end points are : effect of 3 months of oral erythromycin on - pruritus,	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.	at 3 months of treatment	Yes