We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Try the New Site
We're building a modernized ClinicalTrials.gov! Visit Beta.ClinicalTrials.gov to try the new functionality.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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
Sponsor:
Information provided by (Responsible Party):
Del Cont Delphine, Centre Hospitalier Universitaire de Nice

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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.


Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Drug: Oral erythromycin Phase 3

Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 8 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: Oral erythromycin
Oral erythromycin
 Drug: Oral erythromycin
Severe Dowling Meara EBS patients from 6 months to 8 years old


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. 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.

  2. 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.

  3. 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.


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   6 Months to 8 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

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


Locations
Layout table for location information
France
CHU de Dijon Recruiting
Dijon, France
Contact: Pierre Vabres, PU-PH       pierre.vabres@chu-dijon.fr   
Hôpital Saint Eloi Not yet recruiting
Montpellier, France
Contact: Didier Bessis, PH         
CHU de Nice - Hôpital de Cimiez Recruiting
Nice, France, 06000
Contact: Christine Chiaverini, PH    0033 4 92 03 61 07    chiaverini.c@chu-nice.fr   
Contact: Vanina Oliveri, ARC    0033 4 92 03 42 54    oliveri.v@chu-nice.fr   
Hôpital Purpan Recruiting
Toulouse, France
Contact: Juliette MAZEREEUW, PU-PH    00 33 5 67 77 81 41    mazereeuw-hautier.j@chu-toulouse.fr   
Sponsors and Collaborators
Centre Hospitalier Universitaire de Nice
Investigators
Layout table for investigator information
Principal Investigator: Christine Chiaverini, PH Centre Hospitalier Universitaire de Nice
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: Del Cont Delphine, Dr Christine CHIAVERINI, Centre Hospitalier Universitaire de Nice
ClinicalTrials.gov Identifier: NCT01340235    
Other Study ID Numbers: 10-PP-19
First Posted: April 22, 2011    Key Record Dates
Last Update Posted: October 14, 2011
Last Verified: October 2011
Additional relevant MeSH terms:
Layout table for MeSH terms
Epidermolysis Bullosa
Epidermolysis Bullosa Simplex
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Erythromycin
 Erythromycin Estolate
Erythromycin Ethylsuccinate
Erythromycin stearate
Anti-Bacterial Agents
Anti-Infective Agents
Gastrointestinal Agents
Protein Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action