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

This study is currently recruiting participants.
Verified October 2011 by Centre Hospitalier Universitaire de Nice
Sponsor:
Information provided by (Responsible Party):
Del Cont Delphine, Centre Hospitalier Universitaire de Nice
ClinicalTrials.gov Identifier:
NCT01340235
First received: April 11, 2011
Last updated: October 13, 2011
Last verified: October 2011
History of Changes
  Purpose

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 Intervention Phase
Epidermolysis Bullosa
 Drug: Oral erythromycin
 Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Centre Hospitalier Universitaire de Nice:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]
    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:
  • Secondary end points are : effect of 3 months of oral erythromycin on - Global tolerance of treatment. [ Time Frame: at 3 months of treatment ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: Yes ]
    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.


Estimated Enrollment: 8
Study Start Date: June 2011
Estimated Study Completion Date: June 2012
Estimated Primary Completion Date: December 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Oral erythromycin
Oral erythromycin
 Drug: Oral erythromycin
Severe Dowling Meara EBS patients from 6 months to 8 years old

  Eligibility

Ages Eligible for Study:   6 Months to 8 Years
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01340235

Locations
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
Principal Investigator: Christine Chiaverini, PH CHU de NIce
  More Information

No publications provided

Responsible Party: Del Cont Delphine, Dr Christine CHIAVERINI, Centre Hospitalier Universitaire de Nice
ClinicalTrials.gov Identifier: NCT01340235     History of Changes
Other Study ID Numbers: 10-PP-19
Study First Received: April 11, 2011
Last Updated: October 13, 2011
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
France: Committee for the Protection of Personnes

Additional relevant MeSH terms:
Epidermolysis Bullosa
Epidermolysis Bullosa Simplex
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Erythromycin stearate
Erythromycin
 Erythromycin Estolate
Erythromycin Ethylsuccinate
Gastrointestinal Agents
Therapeutic Uses
Pharmacologic Actions
Protein Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Anti-Bacterial Agents
Anti-Infective Agents

ClinicalTrials.gov processed this record on May 16, 2013