E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
This is an open, prospective, controlled case-series documentation to compare intra-individually the efficacy and tolerance of Sericare versus standard treatment in accelerating the epithelialization of skin lesions of patients with Epidermolysis bullosa hereditaria. |
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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 |
Wound re-epithelialization of that part of the skin lesion, which is treated with Sericare (about 50%) compared to that part of the wound, which is covered by non-adhesive wound dressing alone |
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E.2.2 | Secondary objectives of the trial |
• Percentage of wound epithelialization • Touch sensitivity • Pruritus • Exudation • Assessment of efficacy (evaluated by both the investigators and patients or in case of infants one of their parents) • Assessment of tolerance (evaluated by both the investigators and patients or in case of infants one of their parents)
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Patients aged 1-95 years who have provided written informed consent. • EB Patients with skin lesions between 10 and 200 cm2. • prepared and comply with all trial requirements, including the following: o Application of Sericare on one half of the wound at every change of wound dressing; o Regular clinic visits during the treatment period, and follow-up period. • Negative pregnancy test of women of childbearing potential performed within 7 days prior to the start of treatment. • Women of childbearing potential must agree to use an effective method of contraception (Pearl-Index < 1, e.g. hormonal contraception including the combined oral contraceptive pill, the transdermal patch, and the contraceptive vaginal ring, intrauterine devices or sterilization) during treatment and for at least 6 months thereafter. • Men of procreative capacity must agree to use an effective method of contraception during treatment and for at least 6 months thereafter.
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E.4 | Principal exclusion criteria |
• received treatment with systemic steroids during the last 30 days; • uncontrolled diabetes or diabetic ulcers; • diseases or conditions that could, in the opinion of the investigator, interfere with the assessment of safety, tolerance or efficacy; • a skin disorder that is chronic or currently active and which the Investigator considers will adversely affect the healing of the recent wounds or involves the areas to be examined in this case-series documentation; • a history of clinically significant hypersensitivity to the trial medication or surgical dressings to be used in this case-series documentation; • known multiple allergic disorders; • taking, or have taken, any investigational drugs within 3 months prior to the screening visit; • undergoing investigations or changes in management for an existing medical condition; • not likely to complete the case-series documentation for whatever reason.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint for the biometrical evaluation of the therapy is wound re-epithelialization of that part of the wound (about 50%), which is treated with Sericare compared to that part, which is covered by non-adhesive wound dressing alone. Alternatively comparison of 2 comparable wounds (size ≥ 2 cm x 2,5 cm each) is allowed. A blinded assessment of the efficacy is conducted by two independent experts using a chronological series listing the macro-photographs per patient demonstrating for every timepoint, when wound dressings were changed, and a final examination at day 14 (for disease with recent wounds) or day 28 (for disease with delayed wound healing). |
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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 | Yes |
E.7.1.3.1 | Other trial type description |
Case series documentation of orphan disease |
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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 | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
intra-individually controlled and randomized blinded evaluated by 2 experts using the photo document |
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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 | Yes |
E.8.2.3.1 | Comparator description |
intra-individually comparison of Sericare versus non adhesive wound dressing (Mepilex) alone |
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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 | 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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A final visit is planned 14 days after start of treatment for disease with recent wounds or 28 days after start of treatment for disease with delayed wound healing. |
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | 0 |