E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Advanced Hepatocellular Carcinoma (HCC) with Portal Vein Thrombosis (PVT) |
Carcinoma hepatocelular avanzado (CHC) con Trombosis de la Vena Porta (TVP). |
|
E.1.1.1 | Medical condition in easily understood language |
Liver cancer |
Cáncer de hígado |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10019828 |
E.1.2 | Term | Hepatocellular carcinoma non-resectable |
E.1.2 | System Organ Class | 100000004864 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10036206 |
E.1.2 | Term | Portal vein thrombosis |
E.1.2 | System Organ Class | 10019805 - Hepatobiliary 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 |
Assess efficacy and safety of TheraSphere in comparison to standard of care therapy (sorafenib) in the treatment of patients with portal vein thrombosis associated with unresectable hepatocellular carcinoma. |
Evaluar la eficacia y la seguridad de TheraSphere en comparación con el tratamiento de referencia (sorafenib) en el tratamiento de pacientes con trombosis de la vena porta asociada al carcinoma hepatocelular no resecable. |
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E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patients over 18 years of age, regardless of race or gender - Advanced stage HCC with PVT and no extrahepatic metastases - Child-Pugh A - ECOG 0-1 - No contraindication to study treatment or procedures - Not eligible for curative treatment including liver transplant - No prior external beam radiation therapy - No previous treatment with Sorafenib for more than 4 weeks during the 2 previous months; no prior sorafenib-related toxicity - No previous conventional or drug eluting TACE unless it was performed at least 6 months prior to screening phase - No history of organ allograft |
-Tener 18 o más años de edad, independientemente de su sexo -CHC en estadio avanzado con TVP y ausencia de metástasis extrahepática -Child-Pugh A -ECOG 0-1 -Sin contraindicaciones al tratamiento y procedimientos del estudio. -No elegible para tratamiento curativo incluido trasplante hepático -Sin tratamiento previo con radiación de haz externo -Sin tratamiento previo con sorafenib durante más de 4 semanas durante los 2 meses anteriores; sin toxicidad previa relacionada con sorafenib -No haberse sometido previamente a una TACE convencional o liberadora de fármacos excepto si se realizó como mínimo 6 meses antes de la fase de selección -Sin antecedentes de aloinjerto de órgano |
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E.4 | Principal exclusion criteria |
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E.5 End points |
E.5.1 | Primary end point(s) |
Overall Survival |
Supervivencia Global |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
D0 until patient dies |
Desde D0 hasta la muerte del paciente |
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E.5.2 | Secondary end point(s) |
? Time to progression (TTP) based on investigator assessment according to RECIST v 1.1, modified RECIST and EASL response criteria ? Time to worsening of PVT ? Time to symptomatic progression (TTSP) ? Tumor response according to RECIST v 1.1, modified RECIST and EASL response criteria based on investigator evaluations ? Patient reported outcome (PRO) as assessed by the Functional Assessment of Cancer Therapy ? Hepatobiliary Questionnaire (FACT-Hep) questionnaire ? Adverse events |
?Tiempo hasta la progresión del tumor (TTP) basado en la evaluación del investigador de acuerdo con la versión 1.1. de los criterios RECIST, RECIST modificados y criterios de respuesta EASL ?Tiempo hasta el empeoramiento de la TVP ?Tiempo hasta la progresión sintomática (TTSP) ?Respuesta del tumor de acuerdo con la versión 1.1 de los criterios RECIST, RECIST modificados y criterios de respuesta EASL, basada en las evaluaciones del investigador ?Respuesta referida por el paciente (RRP) según la evalúa el cuestionario FACT Hep (cuestionario hepatobiliar para la evaluación funcional del tratamiento antineoplásico) ?Acontecimientos adversos |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
? Time to progression (TTP): D0, W4, W8, W12, W16, W20, W24, WQ8 ? Time to worsening of PVT: D0, W8, W16, W24, WQ8 ? Time to symptomatic progression (TTSP): D0 until ECOG performance status ?2 ? Tumor response: D-14 to 0, W4, W8, W12, W16, W20, W24, WQ8 ? Patient reported outcome: D-14 to 0, W4, W8, W12, W16, W20, W24, WQ8 ? Adverse events: D-14 to 0, W2, W4, W8, W12, W16, W20, W24, WQ8 |
?Tiempo hasta la progresión del tumor (TTP): D0, S4, S8, S12, S16, S20, S24, C8s ?Tiempo hasta el empeoramiento de la TVP: D0, S8, S16, S24, C8s ?Tiempo hasta la progresión sintomática (TTSP): Desde D0 hasta grado de actividad ECOG ?2 ?Respuesta del tumor: D -14 a 0, S4, S8, S12, S16, S20, S24, C8s ?Respuesta referida por el paciente: D -14 a 0, S4, S8, S12, S16, S20, S24, C8s ?Acontecimientos adversos: D -14 a 0, S2, S4, S8, S12, S16, S20, S24, C8s |
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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 | 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 | Yes |
E.8.1.1 | Randomised | Yes |
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 | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
Ensayo de producto sanitario con el IMP sorafenib como comparador |
Medical device trial with the IMP sorafenib being the comparator |
|
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 17 |
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 |
Canada |
Hong Kong |
Korea, Republic of |
Singapore |
Switzerland |
Taiwan |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
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 sujeto (UVUS) |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | |
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 | 4 |