E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Epidermolysis Bullosa Simplex (EBS) |
Epidermolyse bulleuse simple (EBS) |
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E.1.1.1 | Medical condition in easily understood language |
Epidermolysis Bullosa Simplex (EBS) |
Epidermolyse bulleuse simple (EBS) |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10014989 |
E.1.2 | Term | Epidermolysis bullosa |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 primary objective of this study is to evaluate the long-term safety and tolerability of Diacerein 1% Ointment in subjects with EBS that were previously enrolled in studies CCP-020-301 or CCP-020-101. |
Cette étude a pour objectif principal d’évaluer la tolérabilité et l’innocuité à long terme de la pommade à la diacéréine 1 % chez des patients atteints d’EBS ayant déjà participé à l’étude CCP-020-301 ou CCP-020-101. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. In the opinion of the Investigator, the subject is capable of understanding and complying with protocol requirements.
2. The subject or, when applicable, the subject's legally acceptable representative signs and dates a written, informed consent/assent form and any required privacy authorization prior to the initiation of any study procedures.
3. Subject has a documented genetic mutation consistent with EBS.
4. Subject has completed study CCP-020-301 or CCP-020-101 without a major Clinical Safety Report (CSR) reportable protocol deviation.
5. Subject/caregiver agrees to report use of any topical therapies applied to EBS lesions (e.g. medicated cleansers, bleach cleansers, bleach baths, topical antiseptics, topical disinfectants, etc.).
6. If the subject is a woman of childbearing potential, she has a negative urine pregnancy test and agrees to use an approved effective method of birth control, as defined by this protocol, for the duration of the study.
7. Subject is non-lactating and is not planning for pregnancy during the study period.
8. Subject is willing and able to follow all study instructions and to attend all study visits.
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E.4 | Principal exclusion criteria |
1. Subject has EBS lesions to be treated that are infected (i.e., EBS lesions that require topical antibiotic therapy to treat an infection).
2. Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Baseline.
3. The subject was discontinued from the feeder study due to an adverse event judged to be related or possibly related to the study medication. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety will be evaluated in terms of the occurrence of AEs and changes in clinical laboratory parameters, clinical examination findings, vital signs, weight, and urine measurements. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
For each cycle: start day; start day + 8 weeks; start day + 16 weeks |
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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 | No |
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 | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
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 | No |
E.8.2.3 | Other | No |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 8 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Austria |
France |
Germany |
Israel |
Netherlands |
United Kingdom |
United States |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |