E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Epidermolysis bullosa simplex - type Dowling-Meara |
Epdiermolysis bullosa simplex - Typ Dowling-Meara |
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E.1.1.1 | Medical condition in easily understood language |
Butterfly children |
Schmetterlingskinder |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
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 |
Reduction of blister numbers on the treated body surface by 40% in the diacerein group in comparison to placebo within 4 weeks. |
Reduktion der Blasenanzahl an der behandelten Körperfläche um 40% im Vergleich zu Placebo innerhalb von 4 Wochen.
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E.2.2 | Secondary objectives of the trial |
Time until the reaching of the initial blister number (+/-10%) during the follow-up phase. |
Zeitspanne bis zum Wiedererreichen der initialen Blasenzahl (+/-10%) während der Follow-up Phase. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Epidermolysis bullos simplex - type Dowling-Meara
- Mutations in either the keratin 14 or the keratin 5 gene, leading to protein aggregation
- Age: 6-19 |
- Epidermolysis bullosa simplex - Typ Dowling-Meara
- Mutationen im Keratin 14 oder Keratin 5 Gen, die zur Aggregatbildung führen.
- Alter: 6-19 Jahre
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E.4 | Principal exclusion criteria |
- Other types of EB with mutations in the keratin 14 or teh keratin 5 gene.
- participation in another clinical trial at the same time
- known intolerance against diacerein, adjuvants (especially Tartrazine) or other substances with similar structures
- pregnancy or lactation
- relevant other illnesses (acute infections, ...)
- patients with impaired liver functions |
- Andere Formen von EB mit Mutationen in Keratin 14 oder Keratin 5.
- Gleichzeitige Teilnahme an einer anderen klinischen Studie
-Bekannte oder vermutete Unverträglichkeiten gegen Diacerein, Hilfsstoffe des Prüfpräparats (insbesondere Tartrazin) oder Substanzen mit ähnlicher Struktur.
- Schwangerschaft oder Stillzeit (siehe 7.2.2.1)
- Relevante andere Erkrankungen (akute Infektionen, …)
- PatientInnen mit schweren Leberfunktionsstörungen
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E.5 End points |
E.5.1 | Primary end point(s) |
Reduction of blister numbers in the treated area by 40% in comparison to placebo |
Reduktion der Blasenanzahl an den behandelten Körperarealen um 40% im vergleich zu Placebo. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
After 4 weeks |
Nach 4 Wochen |
|
E.5.2 | Secondary end point(s) |
Time span until reaching the initial blister number (+/- 10%). |
Zeitspanne bis zum Erreichen der initialen Blasenzahl (+/- 10%). |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
After 16 weeks. |
Nach 16 Wochen. |
|
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
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 | No |
E.8.7 | Trial has a data monitoring committee | Yes |
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 | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |