Neoplasms

Blood and Lymph Conditions

All Conditions

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Diseases and Abnormalities at or Before Birth

Rare Diseases

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Glioblastoma

Wilms Tumor

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

Wilms' Tumor

All Drugs and Chemicals

Vaccines

WT2725 injection Study drug will be administered every 1-4 weeks

WT2725

Vice President Clinical Development and Medical Affairs, MD

This clinical study is designed to evaluate the safety, immunogenicity and antitumor activity of WT2725. WT2725 will be administered to patients with advanced malignancies known to overexpress WT1

Treatment with other WT1 vaccines in clinical trials has shown evidence of immunogenicity and clinical response in various malignancies.

This study will assist with determining which dose level(s) to use in future clinical studies and will evaluate both clinical and immunological response.

Clinical Study of WT2725 in Patients With Advanced Malignancies

Initial Phase 1 Study of WT2725 in Patients With Advanced Malignancies

Evaluation of the safety and tolerability of WT2725 Dosing Emulsion based on the occurrence of DLT and AEs The safety and tolerability of WT2725 Dosing Emulsion will be evaluated based on the occurrence of DLT and AEs, and the findings from clinical laboratory tests, vital signs measurements, body weight measurements, and electrocardiogram (ECG) results. The incidence of DLT will be evaluated during the DLT Evaluation Period, which extends from the day of the first dose to just prior to the fifth dose of study drug (Days 1 to 29). No more than 4 doses of study drug will be administered during the DLT Evaluation Period.

Tumor response, as evaluated according to irRC, was based on total measurable tumor burden in patients with solid tumors (ie, non-AML patients). Tumor assessments will be conducted during screening, and then every 8 weeks after the first dose of study drug. All tumor assessments may be performed within ±7 days of the scheduled assessment. All patients should complete an End of Study visit within 28 days after the last dose of study drug and prior to the start of alternate antineoplastic therapy.

The modified IWG response criteria were used to assess drug activity in AML patients. Tumor assessments will be conducted during screening, and then every 8 weeks after the first dose of study drug. All tumor assessments may be performed within ±7 days of the scheduled assessment. All patients should complete an End of Study visit within 28 days after the last dose of study drug and prior to the start of alternate antineoplastic therapy.

Sumitomo Pharma Co., Ltd.

Sumitomo Pharma America, Inc.

Serious adverse events (SAEs) will be defined as: • AEs that occurred on or after the first dose of study drug and on or before the 30th day after the last dose of study drug, • AEs with a missing start date and a stop date on or after the first dose of study drug, or • AEs with both a missing start and stop date.

WT2725; injection WT2725: WT2725 injection Study drug will be administered every 1-4 weeks

WT2725 0.3 mg

WT2725 0.9 mg

WT2725 3.0 mg

WT2725 9 mg

WT2725 18 mg

WT2725 27 mg

Total

Age, Categorical

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

Grade Performance Status 0 Fully active, able to carry on all pre-disease performance without restriction

1. Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, eg, light house work, office work
2. Ambulatory and capable of all self care but unable to carry out any work activities. Up and about more than 50% of waking hours
3. Capable of only limited self care, confined to bed or chair more than 50% of waking hours
4. Completely disabled. Cannot carry on any self care. Totally confined to bed or chair
5. Dead

ECOG Performance Status [n(%)] The Eastern Cooperative Oncology Group (ECOG) score

WT2725 Medical Director

Safety Population (defined as all enrolled patients who received at least one dose of study drug.)

Occurrence of Dose-limiting Toxicities and Adverse Events

The MTD could not be established for this study as no DLTs occurred at doses up to and including 27.0 mg

Maximum Tolerated Dose (MTD) of WT2725 Based on the Evaluation of Dose-limiting Toxicity (DLT)

Efficacy irRC population: including all the patients in the Safety population who are not AML patients and whose lesions are measureable and have at least one posttreatment assessment.

Antitumor Responses to WT2725 Based on the Immune-related Response Criteria (irRC)

Efficacy population for modified IWG response: includes all the AML patients in the Safety population who have \>5% marrow blasts or \>50 copy/μｇRNA of quantitative RT-PCR for WT1 transcript before the first date/time of study drug and who have at least one posttreatment assessment.

Immune Response to WT2725

Overall Study

Heart and Blood Diseases

Immune System Diseases

Symptoms and General Pathology

Multiple Myeloma

Neoplasms, Plasma Cell

Lymphoma

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Hematologic Neoplasms

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Lymphoma, Non-Hodgkin

Neoplasms by Histologic Type

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Hematologic Diseases

Hemostatic Disorders

Blood Coagulation Disorders

Vascular Diseases

Paraproteinemias

Blood Protein Disorders

Hemorrhagic Disorders

Myeloproliferative Disorders

Bone Marrow Diseases

Chronic Disease

Disease Attributes

Lymphosarcoma

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Chronic Graft Versus Host Disease

Chronic Myeloid Leukemia

Chronic Myeloproliferative Disorders

OPB-51602 1, 2, 4 and 6 mg/day oral once daily (QD) in a 4 week cycle

OPB-51602

To determine the maximum tolerated dose (MTD) of OPB-51602

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

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

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.

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).

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.

Otsuka Pharmaceutical Co., Ltd.

OPB-51602: 1, 2, 3, 4 and 6 mg/day oral once daily (QD) in a 4 week cycle

OPB-51602 1mg/Day

OPB-51602 2mg/Day

OPB-51602 3mg/Day

OPB-51602 4mg/Day

OPB-51602 6mg/Day

OPB-51602: 1, 2, 3, 4 and 6 mg/day oral once daily (QD) in a 4 week cycle

OPB-51602: once daily during the treatment period

Japan

Leader of Department of "Small Global" Clinical Development

Safety population No statistical analysis provided for Subjects With Treatment Emergent Adverse Events.

Subjects With Treatment Emergent Adverse Events

Efficacy population included all treated subjects who had received at least 1 dose of study drug.

No statistical analysis provided for treatment response.

Treatment Response

OPB-51602 1m/Day

DLT evaluated subjects who had achieved ≧75% study drug compliance during a 4-week (28-day) treatment period starting from Day 4. No statistical analysis provided for Subjects With DLTs.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Clinical Study of WT2725 in Patients With Advanced Malignancies	NCT01621542	Cancer	WT2725	18 Years -	Sumitomo Pharma America, Inc.
Phase I Study of OPB-51602 in Patients With Hematologic Malignancies	NCT01344876	Multiple Myelom... Non-Hodgkin Lym... Acute Myeloid L... Acute Lymphoid ... Chronic Myeloid...	OPB-51602	20 Years - 75 Years	Otsuka Pharmaceutical Co., Ltd.
Clinical Study of WT2725 in Patients With Advanced Malignancies	NCT01621542	Cancer	WT2725	18 Years -	Sumitomo Pharma America, Inc.

Spots Global Cancer Trial Database for acute myeloid leukemia [aml]