E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients > 18 years of age with a documented invasive and/or severe fungal disease that has been intolerant or refractory (rIFI) to Standard of Care (SoC) antifungal treatment. |
Pacientes de una edad superior a 18 años con enfermedad fúngica documentada invasiva y/o grave que sean resistentes o intolerantes al tratamiento antifúngico convencional |
|
E.1.1.1 | Medical condition in easily understood language |
severe fungal diseases who do not easily respond to, or who are intolerant to, the current treatment for their disease. |
Enfermedad fúngica severa que no responde facilmente o que es intolerante al tratamiento para esta enfermedad |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Bacterial Infections and Mycoses [C01] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
•To evaluate the efficacy of SCY-078 as determined by a Data Review Committee by assessing Global Response (composite assessment of clinical, microbiological success) at End of Treatment (EoT). •To evaluate safety of SCY-078. |
•Evaluar la eficacia del SCY-078 según lo determinado por un Comité de Supervisión de Datos evaluando el éxito global (evaluación compuesta del éxito clínico y micológico) al final del tratamiento (EoT) •Evaluar la seguridad del SCY-078 |
|
E.2.2 | Secondary objectives of the trial |
•To evaluate the efficacy of SCY-078 by measuring recurrence of the baseline fungal infection at 42 days after End of Treatment. •To determine subject survival at 42 and 84 days after Day 1 (first dose of study drug). |
• Evaluar la eficacia del SCY-078 midiendo la recidiva de la infección fúngica inicial 42 días después del final del tratamiento • Determinar la supervivencia de los sujetos 42 y 84 días después del Día 1 (primera dosis del fármaco del estudio) |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Subjects must fulfill all of the following criteria to be eligible for study admission: 1. Subject is a male or female adult ≥18 years of age on the day the study informed consent form (ICF) is signed. 2. Subject has a documented eligible acute or chronic invasive candidiasis (including candidemia) and/or acute or chronic severe mucocutaneous candidiasis (excluding VVC) (as defined in Table 1 of the protocol) that is refractory to or intolerant of, or has toxicities associated withat least one approved SoC antifungal treatment. The subject is also eligible if, in the judgement of the investigator, long-term IV antifungal therapy is not feasible or desirable due to clinical or logistical circumstances or if other oral antifungal alternatives are not appropriate. 3. Subject is able to tolerate medication orally or through a nasogastric (NG) tube or percutaneous endoscopic gastrostomy (PEG) tube. (The other criteria are included in the protocol) |
El sujeto debe cumplir los siguientes requisitos: Criterios PRINCIPALES que deben cumplirse para participar en el estudio: 1. El sujeto es un hombre o mujer adulto de una edad superior a 18 años (inclusive) el día en que se firma el formulario de consentimiento fundamentado (FCF) del estudio. 2. El sujeto tiene una candidiasis (incluida candidemia) invasiva aguda o crónica documentada que reúne los requisitos y/o una candidiasis mucocutánea grave aguda o crónica (excluyendo CVV) (tal como se define en la Tabla 1 del protocolo) que es resistente, intolerante o tiene toxicidades asociadas con al menos un tratamiento antifúngico convencional aprobado. El sujeto también es apto si, a juicio del investigador, el tratamiento antifúngico intravenoso a largo plazo no es factible o deseable debido a circunstancias clínicas o logísticas, o si otras alternativas antifúngicas orales no son apropiadas. 3. El sujeto puede tolerar la medicación vía oral o través de una sonda nasogástrica o un tubo de gastrostomía endoscópica percutánea. (El resto de criterios están contemplados en el protocolo) |
|
E.4 | Principal exclusion criteria |
A subject will be excluded from participation in the study if he or she meets any of the following PRINCIPAL exclusion criteria: 1. Subject has an invasive fungal disease with central nervous system, cardiac or eye involvement. 2. Subject has an invasive fungal disease of the bone and/or joint that is expected to require >90 days of study drug treatment. 3. Subject has VVC. Note: Subjects with CMC including vulvovaginal involvement are not excluded. 4. Subject has an inappropriately controlled fungal infection source (e.g., persistent catheters, devices, identified abscess) that is likely to be the source of the fungal infection. 5. Subject is hemodynamically unstable and/or requiring asopressor medication for blood pressure support. 6. Subject has abnormal liver test parameters: AST or ALT >10 x ULN and/or total bilirubin >5 x ULN. Note: Subjects with unconjugated hyperbilirubinemia with a diagnosis of Gilbert’s disease are not excluded. 7. Subject has an absolute neutrophil count <500 neutrophils/μL at Baseline. 8. Subject has a prolonged QTcF interval (Friderica’s correction: QTc = QT / (RR)0.33)>480 ms on the baseline ECG or other abnormalities deemed clinically significant by the investigator that would put the subject at unacceptable risk for participation in the study. (The other criteria are included in the protocol) |
Un sujeto será excluido de la participación en el estudio si cumple cualquiera de los siguientes criterios de exclusión PRINCIPALES: 1. El sujeto tiene una enfermedad fúngica invasiva con afectación del sistema nervioso central, cardíaco u ocular. 2. El sujeto padece una enfermedad fúngica invasiva del hueso y/o articulaciones que exige más de 90 días de tratamiento con el fármaco del estudio. 3. El sujeto presenta candidiasis vulvovaginal (CVV). Nota: Los sujetos con CMC, incluida afectación vulvovaginal no están excluidos del estudio. 4. El sujeto padece una infección micótica controlada por el uso de un dispositivo inadecuado (ej.: catéteres o dispositivos persistentes, abscesos identificados) que puede ser la fuente de la infección fúngica. 5. El sujeto es hemodinámicamente inestable y/o requiere medicación vasopresora para mantener la tensión arterial normal. 6. El sujeto presenta parámetros anormales de prueba hepática: AST o ALT >10 veces el límite superior de normalidad., y/r bilirrubina total superior a 5 veces el límite superior de normalidad. Nota: Los sujetos con hiperbilirrubinemia no conjugada con diagnóstico de enfermedad de Gilbert no están excluidos del estudio 7. El sujeto tiene un número absoluto de neutrófilos <500 neutrófilos/μl en su estado inicial. 8. El sujeto tiene un intervalo QTcF prolongado (corrección de Fridericia: QTc = QT / (RR)0,33 ) > 480 ms en el ECG inicial u otras anomalías clínicamente significativas para el investigador que pondrían al sujeto en un riesgo inaceptable para participar en el estudio. (Los demás criterios están incluidos en el protocolo) |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Primary Endpoints • Efficacy as measured by the percentage of subjects with global success (complete or partial global response) at EoT as determined by the DMC • Safety as measured by: physical examination, vital signs, adverse events (AEs), 12-lead electrocardiogram (ECG) and laboratory tests |
Variables principales: • Eficacia medida por el porcentaje de sujetos con éxito global (respuesta global completa o parcial) al final del tratamiento, según lo determinado por el DMC • Seguridad medida por: examen físico, signos vitales, efectos adversos (EA), electrocardiograma de 12 derivaciones (ECG) y pruebas de laboratorio |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
upto 90days of treatment with SCY-078 |
hasta 90 días de tratamiento con SCY-078 |
|
E.5.2 | Secondary end point(s) |
Efficacy as measured by: • Percentage of subjects with a recurrence of the baseline fungal infection within 42 days after EoT. • Percentage of subjects surviving 42 and 84 days after Day 1 (first dose of study drug) |
Eficacia medida por: • Porcentaje de sujetos con recidiva de la infección fúngica inicial en los 42 días posteriores al final del tratamiento. • Porcentaje de sujetos que sobrevivieron 42 y 84 días después del día 1 (primera dosis del fármaco del estudio) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
42 and 84 days after Day 1 (first dose of study drug) |
42 y 84 días después del día 1 (primera dosis del fármaco del estudio) |
|
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 | 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 |
Austria |
Germany |
Netherlands |
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 | 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 |