Safety and Efficacy of RAD001 + TACE in Localized Unresectable HCC

Phase 2TerminatedNCT01379521

Novartis Pharmaceuticals65 enrolled

Overview

This study will evaluate the role of everolimus in combination with local Transcatheter Arterial Chemoembolization (TACE) procedure in patients with localized unresectable Hepatocellular Carcinoma (HCC).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Hepatocellular Carcinoma

Interventions

everolimusDRUG

everolimus placeboDRUG

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Newly diagnosed hepatocellular carcinoma limited to liver and not suitable for resection, liver transplant, or radiofrequency ablation. * Intermediate stage (stage B) (according to recognized guidelines) and suitable for TACE therapy * At least one nodule between \> 2cm and ≤ 15cm in diameter with no vascular invasion or abdominal lymph node or distant metastases. * Must have 1 tumor which can be measured in 1 dimension according to specified criteria (RECIST and mRECIST) and has not previously been treated with any type of therapy. * ECOG performance status \< 2cm * Cirrhotic status of Child-Pugh class A or early B * HBV-DNA or HBsAg positive at screen or baseline: preventative treatment with anti-viral started 1-2 weeks prior to receiving study drug Exclusion Criteria: * Any local and/or investigational drugs within 28 days prior to randomization * Active bleeding during the last 28 days prior to screening including variceal bleeding * Prior therapy with mTOR inhibitors * Tumor burden of \> 60% liver involvement * Prior systemic or local therapy including TACE except for the first TACE at Day 0), surgery or liver transplantation * Failed first TACE at Day 0, Cycle 1 for any reason * Known history of human immunodeficiency virus (HIV) seropositivity (HIV testing is not mandatory) * Alcohol intake of 80 grams per day * Undergone major surgery ≤ 3 weeks prior to starting study drug or who have not recovered from surgery * Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods Other protocol-defined inclusion/exclusion criteria may apply

Locations (5)

Novartis Investigative Site

Hong Kong, Hong Kong

Novartis Investigative Site

Kaohsiung, Taiwan, Taiwan

Novartis Investigative Site

Kaohsiung City, Taiwan

Novartis Investigative Site

Linkou District, Taiwan

Outcomes

Primary Outcomes

Time to Progression (TTP) Based on the Modified RECIST Criteria

Time to Progression (TTP) defined as the time from the date of randomization to the date of first documented radiological confirmation of disease progression based on modified RECIST criteria. Progressive Disease: \>20% increase in sum of the longest diameters (SLD) of "viable" target lesion (arterial phase enhancement)

Time frame: 3, 6, 12, 18 and 24 months

Secondary Outcomes

Overall Response Rate (ORR) and Disease Control Rate (DCR) Based on the Modified RECIST

Overall response rate was defined as the number of patients whose best overall response was either complete response or partial response according to the modified RECIST. Complete response: Disappearance of arterial phase enhance-ment in all target lesions. Partial response: \>30% decrease in sum of the longest diameters (SLD) of "viable" target lesion (arterial phase enhance-ment)Disease control rate was defined as the number of patients with a best overall response of complete response, partial response or stable disease. The study was terminated early due to slow enrollment of 4 years, higher than anticipated screen failure rate due to a higher than anticipated proportion of patients having advanced liver disease and a change in clinical practice the total of 80 patients was not reached with only 59 patients recruited in total. The study was underpowered due to termination therefore data was not collected and the outcome measure was not analyzed.

Time frame: 6, 12 months, end of study

Time to Progression Based on Original RECIST

Time to Progression (TTP) defined as the time from the date of randomization to the date of first documented radiological confirmation of disease progression based on original RECIST criteria. Progressive Disease: \>20% increase in sum of the longest diameters (SLD) of target lesions with an absolute increase of ≥5 mm; new lesions. The study was terminated early due to slow enrollment of 4 years, higher than anticipated screen failure rate due to a higher than anticipated proportion of patients having advanced liver disease and a change in clinical practice the total of 80 patients was not reached with only 59 patients recruited in total. The study was underpowered due to termination therefore data was not collected and the outcome measure was not analyzed.

Data from ClinicalTrials.gov

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