Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin
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|ClinicalTrials.gov Identifier: NCT01340235|
Recruitment Status : Unknown
Verified October 2011 by Del Cont Delphine, Centre Hospitalier Universitaire de Nice.
Recruitment status was: Recruiting
First Posted : April 22, 2011
Last Update Posted : October 14, 2011
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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.
|Condition or disease||Intervention/treatment||Phase|
|Epidermolysis Bullosa||Drug: Oral erythromycin||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||8 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin|
|Study Start Date :||June 2011|
|Estimated Primary Completion Date :||December 2011|
|Estimated Study Completion Date :||June 2012|
Experimental: Oral erythromycin
Drug: Oral erythromycin
Severe Dowling Meara EBS patients from 6 months to 8 years old
- 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 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.
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|Ages Eligible for Study:||6 Months to 8 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01340235
|CHU de Dijon||Recruiting|
|Contact: Pierre Vabres, PU-PH email@example.com|
|Hôpital Saint Eloi||Not yet recruiting|
|Contact: Didier Bessis, PH|
|CHU de Nice - Hôpital de Cimiez||Recruiting|
|Nice, France, 06000|
|Contact: Christine Chiaverini, PH 0033 4 92 03 61 07 firstname.lastname@example.org|
|Contact: Vanina Oliveri, ARC 0033 4 92 03 42 54 email@example.com|
|Contact: Juliette MAZEREEUW, PU-PH 00 33 5 67 77 81 41 firstname.lastname@example.org|
|Principal Investigator:||Christine Chiaverini, PH||Centre Hospitalier Universitaire de Nice|
|Responsible Party:||Del Cont Delphine, Dr Christine CHIAVERINI, Centre Hospitalier Universitaire de Nice|
|Other Study ID Numbers:||
|First Posted:||April 22, 2011 Key Record Dates|
|Last Update Posted:||October 14, 2011|
|Last Verified:||October 2011|
Epidermolysis Bullosa Simplex
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases, Vesiculobullous
Protein Synthesis Inhibitors
Molecular Mechanisms of Pharmacological Action