A Study of BBI503 in Adult Patients With Advanced Hepatobiliary Cancer

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Brief Title

A Study of BBI503 in Adult Patients With Advanced Hepatobiliary Cancer

Official Title

A Phase II Clinical Study of BBI503 in Adult Patients With Advanced Hepatobiliary Cancer

Brief Summary

      This is an open label, multi-center, phase II study of BBI503 administered to adult patients
      with advanced hepatobiliary cancer who have exhausted all currently approved standard
      anti-cancer treatment options. BBI503 will be administered orally, daily, in continuous
      28-day cycles at a dose of 300 mg once daily. Cycles will be repeated until patients are no
      longer clinically benefiting from therapy.

      Safety, efficacy and tolerability of BBI503 will be assessed for the duration of study

Study Phase

Phase 2

Study Type


Primary Outcome

Disease Control Rate (DCR)

Secondary Outcome

 Objective response rate (ORR)


Hepatocellular Carcinoma



Study Arms / Comparison Groups

Description:  BBI503 will be administered orally, daily, in continuous 28-day cycles at a dose of 300 mg once daily


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

February 2015

Completion Date

December 2020

Primary Completion Date

February 19, 2019

Eligibility Criteria

        Inclusion Criteria:

          -  Signed written informed consent must be obtained and documented according to
             International Conference on Harmonisation (ICH) - Good Clinical Practice (GCP), the
             local regulatory requirements, and permission to use private health information in
             accordance with the Health Insurance Portability and Accountability Act (HIPPA) prior
             to study-specific screening procedures

          -  Histologically or cytologically confirmed hepatocellular carcinoma or
             cholangiocarcinoma, that is metastatic, unresectable, or recurrent; and for which no
             currently approved, standard anti-cancer treatment option is available. Patients must
             have received standard of care treatment prior to enrollment.

          -  ≥ 18 years of age

          -  Measurable disease as defined by Response Evaluation Criteria in Solid Tumors (RECIST)

          -  Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1

          -  Male or female patients of child-producing potential must agree to use contraception
             or avoidance of pregnancy measures during the study and for 30 days after the last
             BBI503 dose

          -  Females of childbearing potential must have a negative serum pregnancy test

          -  Aspartate transaminase (AST) and Alanine transaminase (ALT) ≤ 5.0x the upper limit of
             normal (ULN)

          -  Hemoglobin > 8.0 g/dL

          -  Total bilirubin ≤ 2.5 x ULN

          -  Creatinine ≤ 1.5 x ULN or creatinine clearance > 50 mL/min according to the
             Cockcroft-Gault estimation.

          -  Absolute neutrophil count ≥ 1.5 x 10^9/L

          -  Platelets ≥ 60 x 10^9/L

          -  Life expectancy ≥ 3 months

          -  A patient with hepatocellular carcinoma (HCC) which has arisen out of any medical
             context must also meet the following criteria:

          -  Must not be a candidate for potentially curative resection

          -  Must be Child-Pugh class A or B7 (i.e., in order to be eligible, the total Child-Pugh
             score for a patient must be ≤ 7)

          -  Must have received prior treatment with sorafenib; and have had either disease
             progression during treatment or have had documented intolerance to sorafenib such that
             further treatment with sorafenib is not possible.

          -  Patients with uncontrolled massive ascites or presence of hepatic encephalopathy
             within four (4) weeks of first dose are excluded

          -  A patient with confirmed cholangiocarcinoma of any type must also meet the following

          -  Must have disease which is not amenable to surgical, radiation, or combined modality
             therapy with curative intent

          -  Must have received prior treatment with gemcitabine, either alone or in combination
             with a platinum agent. Patients who are not eligible for gemcitabine must have
             received an alternate first-line systemic chemotherapy regimen

        Exclusion Criteria:

          -  Anti-cancer chemotherapy, radiotherapy, immunotherapy, or investigational agents
             within 7 days of first dose of BBI503. Patients may begin BBI503 on a date determined
             by the investigator and medical monitor for the sponsor provided there is a minimum of
             7 days since last receiving anti-cancer treatment, and that all prior
             treatment-related adverse events (AEs) have resolved or have been deemed irreversible.

          -  Major surgery within 4 weeks prior to first dose (requiring general anesthesia and/or
             inpatient hospitalization for recovery).

          -  Any known symptomatic or untreated brain metastases requiring increase of steroid dose
             within 2 weeks prior to starting on study. Patients with treated brain metastases must
             be stable for 4 weeks after completion of that treatment. Patients must have no
             clinical symptoms from brain metastases and must be either off steroids or on a stable
             dose of steroids for at least 2 weeks prior to protocol enrollment. Patients with
             known leptomeningeal metastases are excluded, even if treated.

          -  Pregnant or breastfeeding

          -  Significant gastrointestinal disorder(s), in the opinion of the treating investigator,
             (e.g., Crohn's disease, ulcerative colitis, extensive gastric and small intestine
             resection); such that absorption of oral medications may be impaired.

          -  Unable or unwilling to swallow BBI503 capsules daily

          -  Uncontrolled intercurrent illness including, but not limited to ongoing or active
             infection, clinically significant non-healing or healing wounds, symptomatic
             congestive heart failure, unstable angina pectoris, cardiac arrhythmia, significant
             pulmonary disease (shortness of breath at rest or mild exertion), uncontrolled
             infection or psychiatric illness/social situations that would limit compliance with
             study requirements (e.g. no reliable transportation).

          -  Subjects with history of another primary cancer, with the exception of: a) curatively
             resected non-melanoma skin cancer; b) curatively treated cervical carcinoma in situ;
             or c) other primary solid tumor with no known active disease present in the opinion of
             the investigator will not affect patient outcome in the setting of current
             hepatobiliary malignancy.

          -  Abnormal ECGs which are clinically significant such as QT prolongation - QTc > 480
             msec, clinically significant cardiac enlargement or hypertrophy, new bundle branch
             block, or signs of active ischemia. Patients with evidence of prior infarction who are
             New York Heart Association (NYHA) functional class II, III, or IV are excluded, as are
             patients with marked arrhythmia such as Wolff Parkinson White pattern or complete
             atrioventricular (AV) dissociation.




18 Years - N/A

Accepts Healthy Volunteers



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Location Countries


Location Countries


Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor

Sumitomo Dainippon Pharma Oncology, Inc

Study Sponsor

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Verification Date

July 2020