Phase I/II Study of OPB-31121 in Patients With Progressive Hepatocellular Carcinoma

Study Overview
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Brief Summary
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The purpose of this study is: Phase1: To evaluate the safety and determine the recommended dose (RD) Phase2: To evaluate the efficacy

Official Title
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A Multicenter, Open-label, Non-randomized, Dose-escalation, Therapeutic Exploratory Trial to Evaluate the Safety and Efficacy of OPB-31121 in Patients With Progressive Hepatocellular Carcinoma

Conditions
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Hepatocellular Carcinoma

Intervention / Treatment
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* Drug: OPB-31121
* Drug: OPB-31121 phase2


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with histopathologically or clinically confirmed diagnosis of hepatocellular carcinoma Patients with Child-Pugh classification A or B Patients unresponsive to standard therapy or for whom standard therapy is intolerable, or for whom there is no appropriate therapy Patients who are able to take oral medication Patients age 20 to 79 years (inclusive) at time of informed consent Patients with an ECOG performance status score of 0-2 Patients have the eligible organ function. Exclusion Criteria: Patients with a primary malignant tumor Patients with a history of liver transplant Patients with brain metastases Patients with a complication of uncontrolled Patients with a psychiatric disorder that might cause difficulty in obtaining informed consent or in conducting the trial

Ages Eligible for Study
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Minimum Age: 20 Years
Maximum Age: 79 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
Allocation: Non-Randomized
Intervention Model: Factorial Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: OPB-31121 p1<br>Phase1 step | Drug: OPB-31121<br>* Oral administration, 400 mg/day or 600 mg once daily after breakfast during the treatment period (1 month)<br>|
| Experimental: OPB-31121 p2<br>Phase2 step | Drug: OPB-31121 phase2<br>* Oral administration, recommended dose from Phase1 once daily after breakfast during the treatment period (6 months)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Subjects With Treatment Emergent Adverse Events | Treatment emergent adverse events observed during outcome measure time frame. | From first study medication to on Day 32 (after repeated 28 days medication from Day 4 to 32) | 
| Number of Participants Who Experienced Dose-Limiting Toxicities (DLTs) | Recommended Dose (RD) of OPB-31121 was defined as the highest dose at which Dose Limited Toxicity (DLT) occurred at an incidence of < 30%. DLT was defined as adverse events related to OPB-31121 occurring until Day 32, and 1) Grade 4 neutrophil count decreased persisting for ≧ 8 days, or Grade 3 or 4 febrile neutropenia, or infection with neutrophil count decreased 2) Grade 4 Plt decreased, or Grade 3 Plt decreased persisting for ≧ 8 days 3) Grade 3 or 4 nausea, vomiting, or diarrhoea that occurred despite the use of an anti-emetic or anti-diarrheal agents 4) Grade 3 or more severe AEsa excluding the AEs presented above 1) to 3) 5) AEs requiring interruption of IMP administration for a period of ≧ 8 consecutive days 6) Same AEs causing interruption of IMP administration twice | From first study medication to on Day 32 (after repeated 28 days medication from Day 4 to 32) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Best Overall Response | Overall response was evaluated based on the Response Evaluation Criteria in Solid Tumors (RECIST guideline) - mRECIST 1.0. | From first dose of study medication up to 28 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hepatocellular carcinoma