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Study of Efficacy and Safety INC280 in Patients With Advanced Hepatocellular Carcinoma

Phase 2
Withdrawn
Conditions
Advanced Hepatocellular Carcinoma
Interventions
Drug: INC280
Drug: Placebo
Registration Number
NCT01964235
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

This study is establish whether INC280 is safe and has beneficial effects in patients with advanced hepatocellular carcinoma known to have dysregulation of c-MET pathway and whose disease progressed while on, or after, treatment with sorafenib or who are intolerant to sorafenib.

Patients will be randomized in a 2:1 ratio to receive INC280 at 600mg BID plus best supportive care (BSC) or placebo plus BSC, until disease progression or intolerable to study treatment. Patients treated with placebo plus BSC will have the opportunity to receive INC280 treatment upon documented further disease progression (RECIST 1.1) per investigator's discretion after unblinding.

Patient will be stratified to geographical region (Asia vs Rest of World ) and tumor burden (present macroscopic vascular invasion and/or extra-hepatic spread vs not present).

Detailed Description

Study was cancelled by Sponsor prior to enrollment of patients.

Recruitment & Eligibility

Status
WITHDRAWN
Sex
All
Target Recruitment
Not specified
Inclusion Criteria
  • Confirmed c-MET pathway dysregulation.- Hepatocellular carcinoma stage B or C according to the Barcelona Clinic Liver cancer staging classification. - Current cirrhotic status of Child-Pugh class A with no encephalopathy. - Documented disease progression during or after discontinuation of sorafenib treatment or intolerance to sorafenib treatment. - Measurable disease as determined by RECIST v1.1. - ECOG performance status ≤ 1
Exclusion Criteria
  • Previous local antineoplastic therapy or investigational drug completed less than 5 half-lives of the agent prior to randomization and have not recovered from clinically significant toxicity from such treatment to grade ≤1 by the NCI-CTCAE. - Received any targeted therapy other than sorafenib.
  • Active bleeding within 28 days prior to screening visit including variceal bleeding (esophageal varices should be treated according to standard practice and procedure completed 28 days prior to screening visit). - Clinically significant venous or arterial thrombotic disease within past 6 months.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
INC280 plus best supportive careINC280Approximately 46 patients will be treated with INC280 600 mg twice a day plus best supportive care.
Placebo plus best supportive carePlaceboApproximately 23 patients will be treated with matching placebo twice a day plus best supportive care.
Primary Outcome Measures
NameTimeMethod
Time to progression using Response Evaluation Criteria In Solid Tumors (RECIST) version 1.1baseline, 6 weeks up to 6 months

Time to progression is the time from the date of baseline evaluation to the date of the first documented radiological confirmation of disease progression.

Secondary Outcome Measures
NameTimeMethod
Overall Response Ratebaseline, every 6 weeks up to 6 months

Overall Response Rate is defined as the proportion of patients with a best overall response of complete response or partial response at any time on study per RECIST version 1.1.

Safety: hematology and chemistry values, vital signs, electrocardiogramsFrom baseline until end of treatment, average 6 months from baseline

Change from baseline values.

Disease Control Ratebaseline, every 6 weeks up to 6 months

Disease control rate is defined as the proportion of patients with a best overall response of complete response, partial response or stable disease at any time on study per RECIST version 1.1.

Safety: adverse events, serious adverse eventsFrom baseline until 30 days post study treatment

Frequency, duration and severity of adverse events.

Overall Survivalrandomization until death, average 10 months

Overall survival is defined as the time from date of randomization to the date of death due to any cause. If a patient is not known to have died, survival will be censored at the date of last contact.

Best Overall Responsedate of treatment, every 6 weeks up to 6 months

Best overall response is defined as the best response recorded from the date of randomization until the date of last tumor assessment per RECIST version 1.1.

Tolerability of study drugFrom date of randomization until end of treatment, average 6 months from baseline

Tolerability will be assessed by summarizing the number of dose interruptions, dose reductions and dose intensity.

Progression Free Survivalrandomization, every 6 weeks up to 6 months

Progression free survival is defined as the time from date of randomization to the date of the first radiologically documented progression or death due to any cause. If a patient has not experienced radiologically documented progression or death, progression free survival is censored at the date of last adequate tumor assessment.

Trial Locations

Locations (3)

Research Medical Center Onc Dept

🇺🇸

Kansas City, Missouri, United States

Novartis Investigative Site

🇨🇭

Genève, Switzerland

Massachusetts General Hospital Mass General Hospital

🇺🇸

Boston, Massachusetts, United States

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