Neoplasms

Heart and Blood Diseases

Blood and Lymph Conditions

Immune System Diseases

All Conditions

Rare Diseases

Multiple Myeloma

Neoplasms, Plasma Cell

Neoplasms by Histologic Type

Hemostatic Disorders

Blood Coagulation Disorders

Vascular Diseases

Paraproteinemias

Blood Protein Disorders

Hematologic Diseases

Hemorrhagic Disorders

Lymphoproliferative Disorders

Immunoproliferative Disorders

Anti-Inflammatory Agents

Antineoplastic Agents

Antiemetics

Gastrointestinal Agents

All Drugs and Chemicals

Dexamethasone

Pomalidomide

Dexamethasone acetate

Glucocorticoids

Hormones

Hormone Antagonists

Antineoplastic Agents, Hormonal

Immunologic Factors

Angiogenesis Inhibitors

Growth Inhibitors

4 mg oral pomalidomide once daily Days 1-21 of each 28-day cycle

40 mg or 20 mg oral dexamethasone once daily on Days 1, 8, 15, 22 of each 28-day cycle

Toru Sasaki, Director

The purpose of this study is to evaluate the efficacy and safety of pomalidomide in combination with dexamethasone in subjects with relapsed and refractory multiple myeloma.

Japanese Phase 2 Study to Evaluate the Efficacy and Safety of Pomalidomide in Subjects With Relapsed and Refractory Multiple Myeloma

A Phase 2, Multicenter, Single-arm, Open-label Study in Japan to Evaluate the Efficacy and Safety of Pomalidomide (CC-4047) in Combination With Dexamethasone in Subjects With Relapsed and Refractory Multiple Myeloma

Myeloma response was defined as a best overall response of stringent complete response (SCR), complete response (CR), very good partial response (VGPR) or partial response (PR) SCR: CR and normal free light chain (FLC) ratio and no clonal cells in bone marrow; CR: Negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and ≤ 5% plasma cells in bone marrow; VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein and urine M-protein level \< 100 mg/24 hours; PR: ≥ 50% reduction of serum M-Protein and reduction in urinary M-protein by ≥ 90% or to \< 200 mg/24 hours. In addition to the above, if present at baseline a ≥ 50% reduction in the size of soft tissue plasmacytomas is also required.

Myeloma response was defined as a best overall response of complete response (CR) or partial response (PR) CR is defined as: - Absence of original monoclonal paraprotein in serum and urine by immunofixation maintained at least 42 days. - \<5% plasma cell in bone marrow aspirate and on bone marrow biopsy, if performed. - No increase in size or number of lytic bone lesions. - Disappearance of soft tissue plasmacytomas. PR requires all of the following: - ≥ 50% reduction in level of serum monoclonal paraprotein, maintained at least 42 days. - Reduction in 24-hour urinary light chain extraction by ≥ 90% or to \< 200 mg, maintained at least 42 days. - For patients with non-secretory myeloma, ≥ 50% reduction in plasma cells in bone marrow aspirate and on biopsy, if performed, for at least 42 days. - ≥ 50% reduction in the size of soft tissue plasmacytomas. - No increase in size or number of lytic bone lesions.

Time to response was calculated as the time from the first dose to the initial documented response (partial response or better) based on IMWG criteria. SCR: CR and normal free light chain (FLC) ratio and no clonal cells in bone marrow; CR: Negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and ≤ 5% plasma cells in bone marrow; VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein and urine M-protein level \< 100 mg/24 hours; PR: ≥ 50% reduction of serum M-Protein and reduction in urinary M-protein by ≥ 90% or to \< 200 mg/24 hours. If present at baseline a ≥ 50% reduction in size of soft tissue plasmacytomas is also required.

Duration of response (calculated for responders only) was defined as time from the initial documented response (partial response or better) to confirmed disease progression, based on IMWG criteria.

PFS was calculated as the time from the first dosing to the first documented progressive disease, as determined by the investigators based on the IMWG Uniform Response criteria, or death, whichever occurred earlier

Relation to study drug was assessed by the Investigator as either suspected or not suspected. Counts represent the suspected relationship. Severity was assessed using National Cancer Institute Common Toxicity Terminology Criteria for Adverse Events version 4.0 (NCI CTCAE v4.0): 1= Mild 2= Moderate 3= Severe 4= Life-threatening and 5= Death related to AE. Serious AEs (SAEs) were those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above. Treatment Emergent Adverse Event (TEAE) was defined as any AE occurring on or after the first treatment of the study medication and within 28 days after the last dose.

Celgene Corporation

Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants \> 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression or pomalidomide discontinuation for any reason.

Pomalidomide Plus Dexamethasone

Pomalidomide: 4 mg oral pomalidomide once daily Days 1-21 of each 28-day cycle Dexamethasone: 40 mg or 20 mg oral dexamethasone once daily on Days 1, 8, 15, 22 of each 28-day cycle

Age, Continuous

≤ 75 Years Old

> 75 Years Old

Age, Customized

Sex: Female, Male

Race (NIH/OMB)

Time from first diagnosis

Stage I

Stage II

Stage III

Stages are: Stage I: Hemoglobin \> 10 g/dL Serum calcium normal On roentgenogram, normal bone structure or solitary bone plasmacytoma only Low M-component production rates (IgG value \< 5 g/dL, IgA value \< 3 g/dL, Bence Jones protein \< 4 g/24h) Stage II: Overall data as minimally abnormal for stage I, and no single value as abnormal as defined for stage III Stage III: 1 or more of below: Hemoglobin \< 8.5 g/dL Serum calcium \> 12 mg/dL Advanced lytic bone lesions (scale 3) High-M-component production rates (IgG value \> 7 g/dL, IgA value \> 5 g/dL, Bence Jones protein \> 12 g/24h)

Durie-Salmon Multiple myeloma staging at screening

0 = (Asymptomatic)

1 = (Symptomatic but completely ambulatory)

2 = (Ambulatory but unable to work)

3 = (Limited self-care)

4 = (Completely disabled)

The ECOG scale is as follows: Grade 0: Fully active, able to carry on all pre-disease activities without restriction; Grade 1: Restricted in physically strenuous activity, ambulatory and able to carry out work of a light nature; Grade 2: Ambulatory and capable of all self-care but unable to work. Up and about more than 50% of waking hours; Grade 3: Capable of only limited self-care, confined to bed or chair \> 50% of waking hours; Grade 4: Completely disabled. Cannot carry on any self-care. Confined to bed or chair.

Eastern Cooperative Oncology Group Performance Status] (ECOG)

The number of prior chemotherapy regimens for myeloma

Prior Chemotherapy Regimens

Prior Stem Cell Transplant for Myeloma

Durie-Salmon Multiple Myeloma staging at screening

Safety Population included all participants who received at least one dose of study medication

Anne McClain, Senior Manager

Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants \> 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.

Efficacy Evaluable Population includes all participants who met eligibility criteria, took at least one dose of study medication, and had a baseline and a post-baseline efficacy assessment.

Myeloma Response Rate Based on the International Myeloma Working Group (IMWG) Uniform Response Criteria

Myeloma Response Rate Based on European Group for Blood and Marrow Transplantation (EBMT) Criteria

Included participants with at least a PR or better based on Assessment using IMWG criteria.

Time to Response

Efficacy Evaluable Population (EEP) includes all participants who met eligibility criteria, took at least one dose of study medication, and had a baseline and a post-baseline efficacy assessment.

Myeloma Response Rate Based on the International Myeloma Working Group (IMWG) Uniform Response Criteria (Later Cut-off Date)

EEP includes all participants who met eligibility criteria, took at least one dose of study medication, and had a baseline and a post-baseline efficacy assessment.

Myeloma Response Rate Based on European Group for Blood and Marrow Transplantation (EBMT) Criteria (Later Cut-off Date)

Included participants with at least a PR or better based on Assessment using IMWG criteria; EPP includes all participants who meet eligibility criteria, take at least one dose of study medication, and have a baseline and a post-baseline efficacy assessment.

Time to Response (Later Cut-off Date)

Duration of Response was not analyzed as there was insufficient data available at Cycle 2, Day 1 of study treatment. There was limited data evaluated and data were not analyzed.

Kaplan-Meier Estimates of Duration of Response

Kaplan-Meier Estimates of Duration of Response (Later Cut-off Date)

Kaplan-Meier Estimates of Progression-free Survival (PFS)

Kaplan-Meier Estimates of PFS (Later Cut-off Date)

Any 1 TEAE

TEAE related to any study drugs

TEAE related to Pomalidomide

TEAE related to Dexamethasone(Dex)

TEAE with ≥ Grade (GR) 3

≥ 1TEAE ≥ GR 3 related to any study drug

TEAE ≥ GR 3 related to Pomalidomide

TEAE ≥ GR 3 related to Dexamethasone

≥ 1 serious TEAE

Serious TEAE related to any study drug

Serious TEAE related to Pomalidomide

Serious TEAE related to Dexamethasone

TEAE leading to stopping of any study drug

TEAE leading to discontinuation of Pomalidomide

TEAE leading to discontinuation of Dexamethasone

Related TEAE leading to stopping of study drug

Related TEAE leading to stopping of Pomalidomide

Related TEAE leading to stopping Dexamethasone

TEAE leading to dose reduction of any study drug

TEAE leading to dose reduction of Pomalidomide

TEAE leading to dose reduction of Dexamethasone

TEA leading to dose interruption of any study drug

TEAE leading to dose interruption of Pomalidomide

TEAE leading to dose interruption of Dexamethasone

Related TEAE leading to dose reduction of any drug

Related TEAE causing a dose reduction Pomalidomide

≥1 related TEAE causing a dose reduction: Dex

Related TEAE leading to interruption of drugs

Related TEAE causing an interruption Pomalidomide

Related TEAE causing an interruption to Dex

TEAE Grade 5

Safety population includes all participants who took at least one dose of study medication.

Number of Participants With Adverse Events

Pomalidomide 4 mg by mouth (PO) daily (QD) on Days 1-21 of each 28-day cycle. Dexamethasone 40 mg PO once daily on Days 1, 8, 15, 22 of each 28-day cycle for those who are ≤ 75 years of age Dexamethasone 20 mg PO once daily on Days 1, 8, 15, 22 of each 28-day cycle for those who are \> 75 years of age

Overall Study

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Japanese Phase 2 Study to Evaluate the Efficacy and Safety of Pomalidomide in Subjects With Relapsed and Refractory Multiple Myeloma	NCT02011113	Multiple Myelom...	Pomalidomide Dexamethasone	20 Years -	Celgene Corporation
Japanese Phase 2 Study to Evaluate the Efficacy and Safety of Pomalidomide in Subjects With Relapsed and Refractory Multiple Myeloma	NCT02011113	Multiple Myelom...	Pomalidomide Dexamethasone	20 Years -	Celgene Corporation

Spots Global Cancer Trial Database for relapsed and refractory multiple myeloma,