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A Study of BBI608 in Combination With Sorafenib, or BBI503 in Combination With Sorafenib in Adult Patients With Hepatocellular Carcinoma

Phase 1
Completed
Conditions
Hepatocellular Carcinoma
Interventions
Registration Number
NCT02279719
Lead Sponsor
Sumitomo Pharma America, Inc.
Brief Summary

This is an open label, three-arm, phase 1 dose escalation study and phase 2 study of BBI608 in combination with sorafenib, or BBI503 in combination with sorafenib. The study population is adult patients with advanced hepatocellular carcinoma who have not received systemic chemotherapy.

Detailed Description

This is an open label, three-arm, phase 1 dose escalation study and phase 2 study of BBI608 in combination with sorafenib, or BBI503 in combination with sorafenib. The study population is adult patients with advanced hepatocellular carcinoma (HCC) who have not received systemic chemotherapy.

The phase 1 portion will involve dose-escalation of BBI608 administered in combination with a fixed starting dose of sorafenib (Arm 1), and dose escalation of BBI503 administered in combination with a fixed starting dose of sorafenib (Arm 2). The fixed starting dose-level of sorafenib for both arms will be 400 mg twice daily (800 mg total daily dose). Eligible patients will be randomized to either Arm 1 or Arm 2.

The phase 2 portion will be an open-label, 3-arm, randomized trial of patients with advanced HCC who have not received prior systemic treatment. Patients will be randomized to receive either, Arm 1: sorafenib administered in combination with BBI608 (at the RP2D determined for BBI608 plus sorafenib during the phase 1 portion); Arm 2: sorafenib in combination with BBI503 (at the RP2D determined for BBI503 plus sorafenib during the phase 1 portion), or Arm 3: sorafenib alone at a starting dose of 400 mg twice daily. The starting dose for sorafenib is the same for all study arms.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
97
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
BBI608 and SorafenibBBI608-
BBI503 and SorafenibBBI503-
BBI608 and SorafenibSorafenib-
BBI503 and SorafenibSorafenib-
SorafenibSorafenib-
Primary Outcome Measures
NameTimeMethod
To Assess the Number of Patients Who Experienced Adverse Events for Phase IB.Adverse events will be assessed at baseline, while the participant is taking drugs, and for 30 days after stopping therapy. It was expected that patients would receive between 4-24 weeks treatment.

Assessment of safety of either BBI608 or BBI503 given in combination with sorafenib to patients with hepatocellular carcinoma by reporting of adverse events and serious adverse events

Assessment of the Dose Limiting Toxicities for the Napabucasin and Amcasertib Arm in Combination With Sorafenib for Phase IB16 weeks including 2-week Sorafenib run-in period prior to initiation of combination therapy

To assess the dose limiting toxicities for phase IB

Determination of the Recommended Phase II Dose for Napabucasin and Amcasertib Arm in Combination With Sorafenib16 weeks including 2-week Sorafenib run-in period prior to initiation of combination therapy

To determine the recommended phase II dose for Napabucasin and Amcasertib arm in combination with sorafenib

Assessment of Disease Control Rate in the Intent to Treat Population- Phase IIAssessment of the preliminary anti-tumor activity by performing tumor assessments at baseline and every 8 weeks after the date of the first dose of protocol therapy

To evaluate the preliminary anti-tumor activity in patients with advanced hepatocellular carcinoma randomized to receive treatment with sorafenib in combination with Napabucasin, sorafenib in combination with Amcasertib\*, or sorafenib alone; Napabucasin and Amcasertib will be administered at their respective RP2D dose levels for combination administration with sorafenib, which were determined during the phase Ib portion of the study.

The radiologic assessments will be evaluated according to Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 and modified RECIST for patients with hepatocellular carcinoma.

Disease control rate (DCR), defined as the proportion of patients with best response of complete response (CR), Partial Response (PR), or stable disease (SD)

Assessment of Objective Response Rate in the Intent to Treat Population - Phase IIAssessment of the preliminary anti-tumor activity by performing tumor assessments at baseline and every 8 weeks after the date of the first dose of protocol therapy

To evaluate the preliminary anti-tumor activity in patients with advanced hepatocellular carcinoma randomized to receive treatment with sorafenib in combination with Napabucasin or sorafenib in combination with Amcasertib, or sorafenib alone; Napabucasin and Amcasertib will be administered at their respective RP2D dose levels for combination administration with sorafenib, which were determined during the phase Ib portion of the study.

The radiologic assessments will be evaluated according to Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 and modified RECIST for patients with hepatocellular carcinoma.

ORR was defined as the percentage of patients with a best overall response (BOR) of complete response (CR) or partial response (PR), using both RECIST and mRECIST assessment data.

Secondary Outcome Measures
NameTimeMethod
Assessment of the Pharmacodynamic Studies as Well as the Concentration of Study Drug (Either Napabucasin or Amcasertib) in TumorsOn day 15 of the second cycle

To perform biomarker studies for napabucasin administered in combination with sorafenib and for amcasertib administered in combination with sorafenib.

Assessment of the Pharmacokinetic Profile (Maximum Plasma Concentration and Area Under the Curve) of Either Napabucasin or Amcasertib.On Day 15 of the first cycle and Day 15 of the second cycle, prior to dosing and every one hours to 11 hours and 24 hours after first dose

To determine the pharmacokinetic (PK) profile of napabucasin administered in combination with sorafenib and of amcasertib administered in combination with sorafenib.

Trial Locations

Locations (29)

USC Norris Comprehensive Cancer Center

🇺🇸

Los Angeles, California, United States

Texas Oncology-Baylor Charles A. Sammons Cancer Center

🇺🇸

Dallas, Texas, United States

Piedmont Cancer Institute, P.C.

🇺🇸

Atlanta, Georgia, United States

Drexel University

🇺🇸

Philadelphia, Pennsylvania, United States

Baptist Health Medical Group Oncology, LLC

🇺🇸

Miami, Florida, United States

Oncology Consultants

🇺🇸

Houston, Texas, United States

Banner MD Anderson Cancer Center

🇺🇸

Gilbert, Arizona, United States

Mayo Clinic

🇺🇸

Phoenix, Arizona, United States

Southern California Research Center

🇺🇸

Coronado, California, United States

The Valley Hospital Luckow Pavilion

🇺🇸

Paramus, New Jersey, United States

Minnesota Oncology Hematology, P.A.

🇺🇸

Minneapolis, Minnesota, United States

USOR - Rocky Mountain Cancer Centers

🇺🇸

Denver, Colorado, United States

California Center Associates for Research and Excellence, Inc. (Ccare)

🇺🇸

Encinitas, California, United States

Winship Cancer Institute of Emory University

🇺🇸

Atlanta, Georgia, United States

USOR - Comprehensive Cancer Centers of Nevada

🇺🇸

Las Vegas, Nevada, United States

Kansas City Research Institure

🇺🇸

Kansas City, Missouri, United States

USOR - New York Oncology Hematology

🇺🇸

Albany, New York, United States

Carolinas Health Care System

🇺🇸

Charlotte, North Carolina, United States

University of Rochester, Wilmot Cancer Institute

🇺🇸

Rochester, New York, United States

Wake Forest Baptist Health

🇺🇸

Winston-Salem, North Carolina, United States

University of Cincinnati, Vontz Center

🇺🇸

Cincinnati, Ohio, United States

USOR - Texas Oncology, Fort Worth 12th Ave

🇺🇸

Fort Worth, Texas, United States

Texas Oncology - El Paso Cancer Treatment Center Joe Battle

🇺🇸

El Paso, Texas, United States

Oncology and Hematology Associates of Southwest Virginia, Inc., DBA Blue Ridge Cancer Care

🇺🇸

Roanoke, Virginia, United States

USOR - Texas Oncology, Tyler

🇺🇸

Tyler, Texas, United States

Texas Oncology-McAllen South Second Street

🇺🇸

McAllen, Texas, United States

Norton Cancer Institute

🇺🇸

Louisville, Kentucky, United States

USOR - Texas Oncology, Austin Midtown

🇺🇸

Austin, Texas, United States

Karmanos Cancer Institute

🇺🇸

Detroit, Michigan, United States

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