E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Dowling Maera's bullous epidermolysis is a génodermatose with autosomique dominant transmission owed to transfers of the genes coding for keratins. It results from it a cutaneous fragility very severe especially during the early childhood. Tetracyclines showed a certain efficiency in cases isolated probably by their anti-inflammatory action but cannot be used at the young child's. The érythromycine, used in the other inflammatory dermatosis, seems to be a good candidate for these patients. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10056508 |
E.1.2 | Term | Acquired epidermolysis bullosa |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The main objective is to estimate the efficiency of the oral érythromycine to decrease the number of cutaneous bubbles at the patients affected by EBS-DM after 3 months of treatment. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives are: - To estimate the efficiency of the oral érythromycine in 3 months for: - Decrease the affected surface, - Decrease the prurit, decrease the cutaneous fragility - To estimate the global tolerance of the treatment. - To estimate the efficiency in term of improvement of the symptomatologie bulleuse, the affected surface and the prurit 2 months after the stop of the treatment, at M5, to estimate the remote preservation of a possible efficiency of the oral érythromycine. - To look for a bacterial colonization by bacteriological takings to M0, M1, M3 and M5, with study of the antibiogramme to detect a possible resistance in the érythromycine as well as its remote preservation.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patient of 2 sexes - Carrier of a severe EBS-DM (more than 2 new bubbles a day on average) - Age from 3 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 by the 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
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E.4 | Principal exclusion criteria |
Patient allergic to the érythromycine - Patient presenting an intolerance to the fructose, a syndrome of bad absorption some glucose and some galactose or a deficit it sucrase-isomaltase - Renal and\or hepatic insufficiency - Patient taking a medicine against indicated or disadvised in association with the erythromycin
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E.5 End points |
E.5.1 | Primary end point(s) |
The main criterion will be patient's rate presenting a decrease of the average number of new cutaneous bubbles a day of at least 20 % after the period of treatment. The number of new bubbles will be counted during a week by the parents on a patient nude, on all the cutaneous surface of the body (including the scalp) before the daily care of drilling of bubbles and bandage. He will be retranscribed in the pad put back(handed) to the patient or to parents. Any new bubble must be counted whatever is its size. A bubble will thus be counted only once, the day of its appearance. The average of the number of new bubbles a day will then be calculated by the investigator. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 4 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 18 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 18 |