E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Epidermolysis Bullosa |
Epidermolisi bollosa |
|
E.1.1.1 | Medical condition in easily understood language |
Genetic skin fragility disorder |
Disordine genetico che caus fragilit¿ della pelle |
|
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. |
dimostrare la sicurezza a lungo termine di ZORBLISA in pazienti affetti da epidermolisi bollosa semplice, distrofica recessiva o giunzionale, di tipo non-Herlitz. |
|
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 |
Gli obiettivi secondari comprendono la valutazione dell'efficacia di ZORBLISA in termini di variazioni dell¿area della superficie corporea (BSA) interessata dalla cute lesa e della portata della ferita, nonch¿ la rimarginazione delle ferite non guarite nel corso dello studio SD-005 |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
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) |
1. Modulo di consenso informato firmato dal soggetto o dal suo rappresentante legale; qualora il soggetto sia minorenne ma in grado di fornire il proprio consenso, dovrà essere ottenuto un modulo di assenso firmato da quest’ultimo. 2. Volontà da parte del soggetto (o del prestatore di cure) di ottemperare a tutti i requisiti del protocollo. 3. Completamento dello studio SD-005 da parte del soggetto (in trattamento con il farmaco in studio alla Visita 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. |
Mancata adesione ai criteri di inclusione di cui sopra. 2. Gravidanza o allattamento nel corso dello studio (per le pazienti in età fertile, verrà eseguito un test di gravidanza sulle urine alla visita finale dello studio SD-005 e sarà ripetuto alla visita 1 se queste visite non avvengono nello stesso giorno). 3. Mancato uso, da parte delle donne in età fertile, di un metodo contraccettivo accettabile, ivi compresa l’astinenza. |
|
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 |
L'endpoint primario dello studio è dimostrare la sicurezza a lungo termine di ZORBLISA in pazienti affetti da epidermolisi bollosa semplice, distrofica recessiva o giunzionale, di tipo non-Herlitz |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Safety is assessed via monitoring of local tolerabilty at the application sites, occurrence of adverse events and physical examinations at Months 1,3,6,9,12,15,18, and 21 |
La sicurezza sarà valutata in base alla tollerabilità locale della crema insorgenza eventi avversi ed esame fisico ai Mesi 1, 3, 6, 9, 12, 15, 18 e 21. |
|
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. |
Gli endpoint secondari comprendono le variazioni dell'area della superficie corporea (BSA) interessata dalla cute lesa e della portata della ferita, nonch¿ la rimarginazione delle ferite non guarite nel corso dello studio SD-005 |
|
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, 21, 24, 27, 30, 33, 36, 39, 42, 45, and 48 compared to Baseline. - Change in total corporal lesions based on BSA estimates at Months 1, 3, 6, 9, 12, 15, 18, 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. The unhealed target wound from SD-005 will be assessed at each subsequent scheduled visit until the target wound is documented as closed. |
Cambiamenti della cute lesa da valutare in base alla BSA ai mesi 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45 e 48 confrontata con il basale. - Cambiamenti nelle lesioni corporee totali da valutare in base alla BSA ai mesi 1, 3 , 6 , 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45 e 48 confrontata con il basale - Per le lesioni bersaglio non rimarginate alla fine dello studio SD-005, l'area delle lesioni bersaglio alla visita finale dello studio SD-005 sar¿ usata come dimensione di lesione basale per lo studio SD-006. Le ferite bersaglio verranno seguite durante ciascuna visita fino alla documentata completa chiusura |
|
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
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 | Information not present in EudraCT |
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 |
Israel |
Serbia |
United States |
Austria |
Belgium |
Bulgaria |
France |
Germany |
Italy |
Lithuania |
Netherlands |
Poland |
Romania |
Spain |
United Kingdom |
|
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
|
LVLS |
Ultima visita dell'ultimo paziente |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 10 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
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 |