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

NCT01340235 | Unknown Phase 3

• 1 other identifier: 10-PP-19
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 4

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.

Conditions

Epidermolysis Bullosa

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (3)

• Secondary end points are : effect of 3 months of oral erythromycin on - Global tolerance of treatment.

  at 3 months of treatment

• Secondary end points are : effect of 3 months of oral erythromycin on - Involved area

  at 3 months of treatment

• Secondary end points are : effect of 3 months of oral erythromycin on - pruritus,

  at 3 months of treatment

Study Arms (1)

Oral erythromycin

EXPERIMENTAL

Oral erythromycin

Drug: Oral erythromycin

Interventions

Oral erythromycin DRUG

Severe Dowling Meara EBS patients from 6 months to 8 years old

Oral erythromycin

Eligibility Criteria

• Age: 6 Months - 8 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Centre Hospitalier Universitaire de Nice: lead

Study Sites (4)

CHU de Dijon

Dijon, France

RECRUITING

Hôpital Saint Eloi

Montpellier, France

NOT YET RECRUITING

CHU de Nice - Hôpital de Cimiez

Nice, 06000, France

RECRUITING

Hôpital Purpan

Toulouse, France

RECRUITING

MeSH Terms

Conditions

Epidermolysis Bullosa

Interventions

Erythromycin

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Intervention Hierarchy (Ancestors)

Macrolides; Polyketides; Lactones; Organic Chemicals

Study Officials

• Christine Chiaverini, PH

  Centre Hospitalier Universitaire de Nice

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Dr Christine CHIAVERINI

Study Record Dates

• First Submitted: April 11, 2011
• First Posted: April 22, 2011
• Study Start: June 1, 2011
• Primary Completion: December 1, 2011
• Study Completion: June 1, 2012
• Last Updated: October 14, 2011

Record last verified: 2011-10

Locations

FR (4)