A Study of BBI503 in Advanced Solid Tumors, or BBI503/ Sorafenib in Advanced Hepatocellular Carcinoma

Study Overview
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Brief Summary
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This is an open-label, multicenter, phase 1 dose escalation study of BBI503 monotherapy, or BBI503 in combination with Sorafenib. This study population is adult patients with advanced solid tumors in monotherapy, or adult patients with advanced hepatocellular carcinoma in combination therapy.

Official Title
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A Phase I Study of BBI503 Monotherapy in Patients With Advanced Solid Tumors, or BBI503 Plus Sorafenib Combination Therapy in Patients With Advanced Hepatocellular Carcinoma

Conditions
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Advanced Solid Tumors, Hepatocellular Carcinoma

Intervention / Treatment
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* Drug: BBI503
* Drug: Sorafenib


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Provision of written informed consent. ≥ 20 years of age Measurable disease as defined by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 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 Adequate organ function Life expectancy ≥ 3 months Exclusion Criteria: Any known symptomatic or untreated brain metastases Pregnant or breastfeeding Crohn's disease, ulcerative colitis extensive gastric and small intestine resection Unable or unwilling to swallow BBI503 daily Uncontrolled concurrent disease Received other investigational drugs within 4 weeks prior to first dose Prior treatment with BBI503

Ages Eligible for Study
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Minimum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BBI503, BBI503 and Sorafenib<br> | Drug: BBI503<br> <br> Drug: Sorafenib<br>* Sorafenib 400mg twice daily (800mg total daily dose)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Determine of the Maximum Tolerated Dose (MTD) of BBI503 monotherapy and in combination with Sorafenib by assessing dose-limiting toxicities (DLTs) |  | 36 days | 
| Number of Participants with Adverse Events as a Measure of Safety and Tolerability |  | Approximately 7 months | 
| Pharmacokinetic profile of BBI503 |  | 37 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assessment of the preliminary anti-tumor activity by performing tumor assessments approximately every 8 weeks | The radiologic assessments will be evaluated according to Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 | 6 months | 
| Progression Free Survival | The time the participant stays on study until progression will be measured and recorded. | Approximately 7 month | 
| Overall Survival | Participants follow-up for overall survival will occur. Maximum follow-up time is 1 year after the initial administration of the last subject. | Approximately1 year |