E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Epidermolysis Bullosa |
Epidermólisis bullosa |
|
E.1.1.1 | Medical condition in easily understood language |
Genetic skin fragility disorder |
enfermedad genética de fragilidad de la piel |
|
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 | 19.0 |
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. |
El objetivo principal es demostrar la seguridad a largo plazo de ZORBLISA en pacientes con epidermólisis ampollosa simple, distrófica recesiva y juntural tipo no-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 |
Los objetivos secundarios son evaluar la eficacia de ZORBLISA en cuanto a los cambios en la superficie corporal (SC) afectada por lesiones y en la carga asociada a las heridas, así como el cierre de las heridas no cicatrizadas durante el estudio SD-005. |
|
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) |
1. Consentimiento informado firmado por el sujeto o por el representante legal del sujeto; si el sujeto es menor de 18 años de edad pero es capaz de dar su asentimiento, asentimiento firmado por el sujeto. 2. El sujeto (o su cuidador) debe estar dispuesto a cumplir todos los requisitos del protocolo. 3. Pacientes que hayan completado el estudio SD-005 (con administración del fármaco del estudio en la 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.) 3. Females of childbearing potential who are not abstinent or not practicing a medically acceptable method of contraception. |
1. Pacientes que no cumplan los criterios de inclusión especificados con anterioridad. 2. Embarazo o lactancia durante el estudio. (En el caso de las pacientes en edad fértil, se realizará una prueba de embarazo en orina en la visita final del estudio SD-005). 3. Mujeres en edad fértil que no se abstengan de tener relaciones sexuales o no empleen un método de anticoncepción aceptable |
|
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 |
El criterio principal de valoración es la seguridad a largo plazo de ZORBLISA en pacientes con epidermólisis ampollosa simple, distrófica recesiva y juntural tipo no-Herlitz. |
|
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 |
La seguridad se evaluará mediante monitorización de la tolerabilidad a nivel local, acontecimientos adversos y exploraciones físicas los meses 1, 3, 6, 9 y 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. |
los objetivos secundarios incluyen el cambio desde basal en la cobertura SC de las lesiones y el nivel de lesiones, así como el cierre de lesiones diana que no han curado desde el ensayo 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 and 12 compared to Baseline - Change in total body wound coverage based on BSA estimates at Months 1, 3, 6, 9 and 12 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. |
- Cambio de la piel afectada en base a los cálculos de SC en los meses 1, 3, 6, 9 y 12 en coparación con el periodo basal - Cambio en la cobertura de la superficie corporal afectada por lesiones, en base a estimaciones de la SC en los meses 1, 3, 6, 9 y 12 en coparación con el periodo basal - Para lesiones diana que no habían cerrado en el estudio SD-005, el area de la lesión diana en la visita final del estudio SD-005 se usará como basal para el estudio SD-006. Las lesiones diana se evaluarán en cada visita hasta que se documente el cierre |
|
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.8.5 | 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 |
Austria |
France |
Germany |
Italy |
Netherlands |
Poland |
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
|
LVLS |
Última visita del último paciente |
|
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 | |
E.8.9.2 | In all countries concerned by the trial years | 2 |