E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
advanced hepatocellular carcinoma with ChildPugh liver function class B |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10024720 |
|
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 compare two doses of sorafenib (800 and 400 mg/day) with best supportive care in terms of survival in HCC patients with impaired liver function (Child B). |
|
E.2.2 | Secondary objectives of the trial |
to compare the three treatment arms in terms of toxicity and quality of life. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
� Patients with diagnosis of HCC according to the AASLD- EASL criteria (10) � Age >18 years � Advanced stage of disease defined as HCC not eligible for locoregional treatments (ab initio or following progression after such treatments) � Liver function classified as Child class B � ECOG performance status � 2 � Adequate renal (creatinine � 1.5 x ULN), and haematological function (platelet count � 60 x 10^9/L, hemoglobin � 9 g/dl) � Life expectancy of at least 2 months � Written informed consent |
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E.4 | Principal exclusion criteria |
� Prior exposure to sorafenib � Performance Status (ECOG) �3 � Concomitant diseases that contraindicates the use of sorafenib � Gastro-intestinal bleeding in the previous 30 days � Serious active infections (> grade 2 CTCAE version 3.0) � Pregnant or lactating women � Not cooperative patients � Inability of patients to access to the hospital � Grade 3-4 encephalopathy that makes the patient not able to follow study procedures (Table 2) � Refusal of informed consent |
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E.5 End points |
E.5.1 | Primary end point(s) |
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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 |
sola terapia di supporto - same IMP used at different dosage |
|
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 | 40 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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
|
L`analisi statistica finale potra` essere effettuata al raggiungimento di 340 eventi complessivi nei tre bracci di studio. |
|
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 | 0 |
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 | 0 |