E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
Genetic skin fragility disorder |
|
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 |
|
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 is to demonstrate the long-term safety of ZORBLISA in patients, with Simplex, Recessive Dystrophic and Junctional non-Herlitz Epidermolysis Bullosa. |
|
E.2.2 | Secondary objectives of the trial |
The secondary objectives are to assess the efficacy of ZORBLISA in terms of the change in Body Surface Area (BSA) of lesional skin and wound burden; as well as closure of unhealed target wounds from the SD-005 study |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Informed Consent form signed by the patient or patient's legal representative; if the patient is under the age of 18 but capable of providing assent, signed assent from the patient. 2. Patient (or caretaker) must be willing to comply with all protocol requirements. 3. Patient who completed the SD-005 study (on study drug at Visit 5) |
|
E.4 | Principal exclusion criteria |
1. Patients who do not meet the entry criteria outlined in the Inclusion criteria. 2. Pregnancy or breastfeeding during the study. (A urine pregnancy test will be performed at the final visit for SD-005 for female patients of childbearing potential and repeated at Visit 1 if these visits do not occur on the same day) 3. Females of childbearing potential who are not abstinent or not practicing a medically acceptable method of contraception. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the long-term safety of ZORBLISA in patients with Simplex, Recessive Dystrophic, and Junctional non-Herlitz Epidermolysis Bullosa |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Safety is assessed via monitoring of local tolerability at the application sites, occurrence of adverse events and physical examinations at Months 1, 3, 6, 9 and 12 |
|
E.5.2 | Secondary end point(s) |
The secondary endpoints include the change from baseline in BSA coverage of lesional skin and wound burden; as well as closure of unhealed target wounds from the SD-005 study. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
- Change in lesional skin based on BSA estimates at Months 1, 3, 6, 9, 12, 15, 18, and 21, 24, 27, 30, 33, 36, 39, 42, 45, and 48 compared to Baseline - Change in total body wound coverage based on BSA estimates at Months 1, 3, 6, 9, 12, 15, 18, and 21, 24, 27, 30, 33, 36, 39, 42, 45, and 48 compared to Baseline - For target wounds that are not closed by the end of Study SD-005, the target wound area at the final visit for Study SD-005 will be used as the baseline area size of the target wound for SD-006. Target wounds will be assessed at each subsequent visit until the wound is documented as closed. |
|
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 | No |
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 | 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.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
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 |
Bulgaria |
France |
Germany |
Israel |
Italy |
Lithuania |
Netherlands |
Poland |
Romania |
Serbia |
Spain |
United Kingdom |
United States |
|
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
|
|
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 | 48 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 48 |