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

This study has been completed.
Information provided by (Responsible Party):
Otsuka Pharmaceutical Co., Ltd. Identifier:
First received: July 28, 2011
Last updated: May 21, 2015
Last verified: May 2015

The purpose of this study is:

Phase1: To evaluate the safety and determine the recommended dose (RD) Phase2: To evaluate the efficacy

Condition Intervention Phase
Hepatocellular Carcinoma
Drug: OPB-31121
Drug: OPB-31121 phase2
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Factorial Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Further study details as provided by Otsuka Pharmaceutical Co., Ltd.:

Primary Outcome Measures:
  • Subjects With Treatment Emergent Adverse Events [ Time Frame: From first study medication to on Day 32 (after repeated 28 days medication from Day 4 to 32) ]
    Treatment emergent adverse events observed during outcome measure time frame.

  • Number of Participants Who Experienced Dose-Limiting Toxicities (DLTs) [ Time Frame: From first study medication to on Day 32 (after repeated 28 days medication from Day 4 to 32) ]

    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

Secondary Outcome Measures:
  • Best Overall Response [ Time Frame: From first dose of study medication up to 28 weeks ]
    Overall response was evaluated based on the Response Evaluation Criteria in Solid Tumors (RECIST guideline) - mRECIST 1.0.

Enrollment: 23
Study Start Date: July 2011
Study Completion Date: March 2014
Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: OPB-31121 p1
Phase1 step
Drug: OPB-31121
Oral administration, 400 mg/day or 600 mg once daily after breakfast during the treatment period (1 month)
Experimental: OPB-31121 p2
Phase2 step
Drug: OPB-31121 phase2
Oral administration, recommended dose from Phase1 once daily after breakfast during the treatment period (6 months)


Ages Eligible for Study:   20 Years to 79 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01406574

Chiba, Japan
Tokyo, Japan
Sponsors and Collaborators
Otsuka Pharmaceutical Co., Ltd.
  More Information

Responsible Party: Otsuka Pharmaceutical Co., Ltd. Identifier: NCT01406574     History of Changes
Other Study ID Numbers: 252-11-001
JapicCTI-111546 ( Other Identifier: JAPIC )
Study First Received: July 28, 2011
Results First Received: April 14, 2015
Last Updated: May 21, 2015

Keywords provided by Otsuka Pharmaceutical Co., Ltd.:
Hepatocellular carcinoma

Additional relevant MeSH terms:
Carcinoma, Hepatocellular
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Liver Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Liver Diseases processed this record on May 23, 2017