MedDataX Logo

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

NCT ID: NCT01340235

Last Updated: 2011-10-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Clinical Phase

PHASE3

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-06-30

Study Completion Date

2012-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Epidermolysis Bullosa

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Oral erythromycin

Oral erythromycin

Group Type EXPERIMENTAL

Oral erythromycin

Intervention Type DRUG

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Oral erythromycin

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

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

6 Months

Maximum Eligible Age

8 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Centre Hospitalier Universitaire de Nice

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Del Cont Delphine

Dr Christine CHIAVERINI

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Christine Chiaverini, PH

Role: PRINCIPAL_INVESTIGATOR

Centre Hospitalier Universitaire de Nice

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

CHU de Dijon

Dijon, , France

Site Status RECRUITING

Hôpital Saint Eloi

Montpellier, , France

Site Status NOT_YET_RECRUITING

CHU de Nice - Hôpital de Cimiez

Nice, , France

Site Status RECRUITING

Hôpital Purpan

Toulouse, , France

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

France

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Pierre Vabres, PU-PH

Role: primary

[email protected]

Didier Bessis, PH

Role: primary

Christine Chiaverini, PH

Role: primary

[email protected]
0033 4 92 03 61 07

Vanina Oliveri, ARC

Role: backup

[email protected]
0033 4 92 03 42 54

Juliette MAZEREEUW, PU-PH

Role: primary

[email protected]
00 33 5 67 77 81 41

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

10-PP-19

Identifier Type: -

Identifier Source: org_study_id