Clinical trial NCT01003015: Safety Study of BAY73-4506 in Patients With Hepatocellular Carcinoma
Official title: An Uncontrolled Open Label Multicenter Phase II Safety Study of BAY73-4506 in Patients With Hepatocellular Carcinoma (HCC)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Carcinoma, Hepatocellular
Keywords: HCC
Safety
BAY73-4506
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2009-10-28
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine whether BAY73-4506 treatment is safe and can shrink or delay the growth of tumors in patients with unresectable liver cancer.


Eligibility criteria: Inclusion Criteria:

* Male or female patients aged equal or above 18 years.
* BCLC stage Category A, B or C that cannot benefit from treatments of established efficacy with higher priority such as resection, liver transplantation, local ablation, chemoembolization or systemic sorafenib.
* Liver function status Child-Pugh class A.
* Failure to prior treatment with sorafenib (defined as radiological progression under sorafenib therapy)
* Local or loco-regional therapy (eg, surgery, radiation therapy, hepatic arterial embolization, chemoembolization, radiofrequency ablation, percutaneous ethanol injection, or cryoablation) must have been completed = 4 weeks before first dose of BAY73-4506.
* ECOG PS of 0 or 1.
* Adequate bone marrow, liver and renal function

Exclusion Criteria:

* Prior systemic treatment with molecular targeted agents for HCC, except sorafenib. Prior chemotherapy treatment is allowed.
* Known history or symptomatic metastatic brain or meningeal tumors (head CT or MRI at screening to confirm the absence of central nervous system \[CNS\] disease if patient has symptoms suggestive or consistent with CNS disease).
* Congestive heart failure NYHA\>/= class 2
* Unstable angina (angina symptoms at rest, new onset angina within the last 3 months) or myocardial infarction (MI) within the past 6 months before start of study medication.
* Cardiac arrhythmias requiring anti-arrhythmic therapy (beta blockers or digoxin are permitted).
* Uncontrolled hypertension (systolic blood pressure \> 150 mmHg or diastolic pressure \> 90 mmHg despite optimal medical management).
* Arterial or venous thrombotic or embolic events such as cerebrovascular accident (including transient ischemic attacks), deep vein thrombosis or pulmonary embolism within 6 months before the start of study treatment.

Primary outcomes:
measure: Adverse Event Collection
timeFrame: Up to 30+/- 7 days after permanently discontinuing BAY73-4506 administration
Secondary outcomes:
measure: Time to progression
timeFrame: Every 6 weeks during treatment and after 6 cycle treatment every 18 weeks till progression
measure: Objective response rate
timeFrame: Every 6 weeks during treatmen and after 6 cycle treatment every 18 weeks till progression
measure: Disease control rate
timeFrame: Every 6 weeks during treatmen and after 6 cycle treatment every 18 weeks till progression
measure: Overall survival
timeFrame: Every 6 weeks during treatmen and after 6 cycle treatment every 18 weeks till progression
measure: Trough concentration of Regorafenib and metabolites (for Europe only)
timeFrame: Cycle 1 Day 15 and Cycle 2 Day 1
measure: Full Pharmacokinetics profile of BAY73-4506 and metabolites (for Korea only)
timeFrame: Cycle 1 Day 21 to Day 28
Other outcomes:
Locations:
city: Regensburg
country: Germany
state: Bayern
city: Frankfurt
country: Germany
state: Hessen
city: Essen
country: Germany
state: Nordrhein-Westfalen
city: Mainz
country: Germany
state: Rheinland-Pfalz
city: Magdeburg
country: Germany
state: Sachsen-Anhalt
city: Rozzano
country: Italy
state: Milano
city: Bologna
country: Italy
city: Milano
country: Italy
city: Milano
country: Italy
city: Roma
country: Italy
city: Daegu
country: Korea, Republic of
city: Seoul
country: Korea, Republic of
city: Barcelona
country: Spain
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References:
citation: Bruix J, Tak WY, Gasbarrini A, Santoro A, Colombo M, Lim HY, Mazzaferro V, Wiest R, Reig M, Wagner A, Bolondi L. Regorafenib as second-line therapy for intermediate or advanced hepatocellular carcinoma: multicentre, open-label, phase II safety study. Eur J Cancer. 2013 Nov;49(16):3412-9. doi: 10.1016/j.ejca.2013.05.028. Epub 2013 Jun 25.