Phase I Study of OPB-51602 in Patients With Hematologic Malignancies

Study Overview
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Brief Summary
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To determine the maximum tolerated dose (MTD) of OPB-51602

Official Title
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A Dose-escalation Trial to Investigate the Safety and Tolerability of OPB-51602 in Patients With Relapsed or Refractory Hematologic Malignancies (Phase 1)

Conditions
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Multiple Myeloma, Non-Hodgkin Lymphoma, Acute Myeloid Leukemia, Acute Lymphoid Leukemia, Chronic Myeloid Leukemia

Intervention / Treatment
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* Drug: OPB-51602


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with a confirmed diagnosis of MM, NHL, AML, ALL or CML. Patients who are responsive or have relapsed following standard treatment Patients capable of providing written informed consent Japanese patients age 20 to 75 years (inclusive) at time of informed consent ECOG performance status score of 0-1 Life expectancy of at least 3 months Adequate vital organ function Patients who, together with their partner, are willing and capable of using an appropriate method of contraception throughout the trial period and until at least 12 weeks after final IMP administration Exclusion Criteria: Patients with other primary malignant tumors Symptomatic CNS involvement Ongoing or active infection, or complication that is not controllable by medication or other means Complication of uncontrolled cardiac disease Female patients who are pregnant, possibly pregnant, or lactating, or who wish to become pregnant during the study period Patients who have received another study drug, or who have received chemotherapy, immunotherapy, cytokine therapy, surgery, or radiotherapy for treatment of the primary disease, within 4 weeks prior to enrollment

Ages Eligible for Study
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Minimum Age: 20 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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Male

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: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: OPB-51602<br>OPB-51602 1, 2, 4 and 6 mg/day oral once daily (QD) in a 4 week cycle | Drug: OPB-51602<br>* once daily during the treatment period<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. A Treatment Emergent Adverse Event was defined as an AE occurring after the start of IMP administration. | From first study medication to on Day 31 (after repeated 28 days medication from Day 4 to 31) | 
| Number of Participants Who Experienced Dose-Limiting Toxicities (DLTs) | DLT was defined as adverse events occurring during Cycle 1 and: (1) Grade 3 or higher nausea, vomiting, or diarrhea despite the use of anti-emetic or antidiarrheal drugs, (2) Grade 3 or higher non-hematologic toxicity, excluding alopecia, (3) AEs requiring interruption of the IMP for a total of 8 days or longer, (4) Grade 4 neutropenia lasting ≥ 8 days (not applicable for leukemia), (5) Grade 3 or higher febrile neutropenia or infection due to neutropenia (not applicable for leukemia), (6) Grade 4 thrombocytopenia or Grade 3 thrombocytopenia requiring platelet transfusion (not applicable for leukemia). | From first study medication to on Day 31 (after repeated 28 days medication from Day 4 to 31) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment Response | Assessment of the treatment response was evaluated according to internationally recognized response criteria for multiple myeloma, non-Hodgkin's lymphoma, acute myeloid leukemia, chronic myeloid leukemia. Response was defined as at least partial response or partial remission (PR) according to the criteria for efficacy assessment. | From first dose of study medication to withdrawal examination | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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multiple myeloma [MM], non-Hodgkin lymphoma [NHL], acute myeloid leukemia [AML], acute lymphoid leukemia [ALL], chronic myeloid leukemia [CML]