A Study of Lenalidomide Versus Placebo in Subjects With Transfusion Dependent Anemia in Lower Risk Myelodysplastic Syndrome (MDS) Without Del 5q
- Registration Number
- NCT01029262
- Lead Sponsor
- Celgene
- Brief Summary
The purpose of this study is to investigate whether lenalidomide would reduce the number of red blood cell transfusions (RBC) needed in anemic (RBC transfusion-dependent) participants with low or intermediate-1 risk MDS without a deletion 5q chromosome abnormality. The study also investigated the safety of lenalidomide use in these participants. Two-thirds of the participants received oral lenalidomide and one-third of the participants received oral placebo.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 239
- 18 years or older
- Diagnosis of low or intermediate-1 risk Myelodysplastic (MDS) with any chromosome karyotype except del 5q[31]
- Anemia that requires red blood cell transfusions
- Resistant to erythropoiesis stimulating agents (ESAs) or blood erythropoietin level > 500 mU/mL
- Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) ≤ 2
- Must agree to follow pregnancy precautions as required by the protocol.
- Must agree to receive counseling related to teratogenic and other risks of lenalidomide
- Must agree not to donate blood or semen
- Must be willing to consent to two or more bone marrow aspirate procedures to be completed during study
- Subjects previously receiving immunomodulating or immunosuppressive agents, or epigenetic or deoxyribonucleic acid (DNA) modulation agents
- Allergic reaction to thalidomide
- Renal insufficiency creatinine clearance (CrC1)<40 mL/min by Cockcroft-Gault method)
- Prior history of cancer, other than MDS, unless the subject has been free of the disease for ≥ 5 years. (Basal cell carcinoma of the skin, carcinoma in situ of the cervix, or stage Tumor (T) 1a or T1b prostate cancer is allowed)
- Absolute neutrophil count (ANC) < 500/uL
- Platelets < 50,000/uL
- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3X upper limit of normal
- Uncontrolled hyperthyroidism or hypothyroidism
- Significant neuropathy
- Prior stem cell transplantation
- Anemia due to reasons other than MDS
- History of deep venous thrombosis (DVT) or pulmonary embolus (PE) within past 3 years
- Significant active cardiac disease within the past 6 months
- Known Human Immunodeficiency Virus (HIV) infection; known Hepatitis C infection or active Hepatitis B infection
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Arm #2 - placebo Placebo Three placebo capsules once daily for at least 168 days until disease progression occurred, intolerable side effects or withdrawal of consent. Arm #1 - Lenalidomide plus placebo Lenalidomide Lenalidomide 10 mg by mouth (PO) daily plus 2 placebo capsules for participants with a creatinine clearance ≥ 60 mL/min for at least 168 days until disease progression, intolerable side effects or withdrawal of consent. Lenalidomide 5 mg PO daily plus 2 placebo capsules for participants with a creatinine clearance ≥ 40 and \< 60 mL/min.
- Primary Outcome Measures
Name Time Method Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for ≥ 56 Days as Determined by an Independent Review Committee (IRC) From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively. The percentage of participants who achieved the 56-day RBC transfusion independent (TI) response was defined as the absence of any RBC transfusions during any consecutive "rolling" 56-day interval within the double-blind treatment phase (ie, Days 2 (Day 1 is the first study drug day) to 57, Days 3 to 58, etcetera). The double-blind treatment phase was defined as the period between the 1st dosing up until 28 days after the last study drug dose
Percentage of Participants With a Erythroid Gene Signature Who Achieved RBC Transfusion Independence for ≥ 56 Days as Determined by an Independent Review Committee (IRC) From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively. The percentage of participants who achieved the 56-day RBC TI response was defined as the absence of any RBC transfusions during any consecutive "rolling" 56-day interval within the double-blind treatment phase (ie, Days 2 (Day 1 is the first study drug day) to 57, Days 3 to 58, etcetera). A participant who achieved at least a 56-day RBC-transfusion-independent response was considered a 56-day RBC-TI responder.
- Secondary Outcome Measures
Name Time Method Percentage of Participants Who Achieved an Erythroid Response Based on the Modified International Working Group (IWG) 2006 Criteria From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively. A participant was considered as having achieved an erythroid response if the participant either:
- had a hemoglobin (Hgb) increase ≥1.5 g/dL compared to baseline and confirmed by another central laboratory hemoglobin value at 4 to 8 weeks after the first Hgb measurement that also increased ≥1.5 g/dL. All Hgb values during this time interval must have had a ≥ 1.5 g/dL increase (ie, no central laboratory Hgb increase during this timeframe could be less \<1.5 g/dL) OR - had a 50% reduction in the number of the RBC transfusion units over any consecutive 56 days period compared to the baseline transfusion burden.
The baseline transfusion burden is the number of units over 112 days by the randomization divided by 2. Only transfusions given for a pre-transfusion Hgb value of 9 g/dL or less were used in this response assessment.Number of Participants With Treatment Emergent Adverse Events (TEAE) From the first dose of study drug through 28 days after discontinuation from the study treatment; up to the final data cut-off date of 03 July 2018; maximum exposure was 2100 days in the lenalidomide arm and 529 days in the placebo arm. A TEAE was defined as an AE that begins or worsens in intensity of frequency on or after the first dose of study drug through 28 days after last dose of study drug.
A serious adverse event (SAE) is any:
* Death;
* Life-threatening event;
* Any inpatient hospitalization or prolongation of existing hospitalization;
* Persistent or significant disability or incapacity;
* Congenital anomaly or birth defect;
* Any other important medical event
The investigator determined the relationship of an AE to study drug based on the timing of the AE relative to drug administration and whether or not other drugs, therapeutic interventions, or underlying conditions could provide a sufficient explanation for the event. The severity of an AE was evaluated by the investigator according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) (Version 3.0) where Grade 1 = Mild, Grade 2 = Moderate, Grade 3 = Severe, Grade 4 = Life-threatening and Grade 5 = Death.Percentage of Participants Who Achieved RBC Transfusion Independence With a Duration of ≥ 24 Weeks (168 Days) as Determined by the Sponsor From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively. The 168-day RBC-transfusion-independent response was defined as the absence of any RBC transfusion during any consecutive "rolling" 168 days during the treatment period, for example Days 2 (Day 1 is the first study drug day) to 169, Days 3 to 170, Days 4 to 171, etcetera. A responder was defined as a participant who had a ≥ 168 consecutive days of RBC-transfusion-free period after the first dose of study drug in the treatment phase.
Kaplan Meier Estimates of Duration of 56-day RBC Transfusion Independence Response as Determined by the Sponsor Response was assessed up to the end of treatment; up to the data cut-off date of 17 Mar 2014. The duration of the first 56-day RBC transfusion-independence response was calculated for those who achieved a response and was dependent on whether a subsequent RBC transfusion was given after the transfusion-free period (response):
* For those who received a subsequent RBC transfusion after the response starts, the duration of response was not censored, and was calculated as response duration = last day of response - first day of response +1 where the last day of response was defined as 1 day before the first RBC transfusion which was given at 56 days or more after the response starts.
* For those who did not receive a subsequent RBC transfusion after the response started, the end day of the response was censored and duration of the response was calculated as response duration = date of last RBC transfusion assessment - first day of response+ 1. A responder was a participant who had a ≥ 56 consecutive days of RBC-transfusion-free period after the first study drug treatment periodTime to 56-Day RBC-Transfusion-Independent (TI) Response as Determined by the Sponsor From first dose of study drug until 28 days after the last dose of study drug, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively. The time to the first 56-day RBC-transfusion-independent response was calculated for participants who achieved a response. The day from the first dose of study drug to the date at which RBC-transfusion-independence starts was achieved and calculated using: Start date of the first response period - the date of the first study drug +1. A responder was defined as a participant who had a ≥ 56 consecutive days of RBC-transfusion-free period after the first dose of study drug in the treatment phase.
Kaplan Meier Estimates for Progression to Acute Myeloid Leukemia (AML) From randomization to final data cut-off date of 03 Jul 2018; median follow up time for progression to AML was 2.3 years (range = 0 to 5.0 years) in the placebo arm and 2.6 years (range = 0 to 6.4 years) in the lenalidomide arm. Progression to AML is part of the natural course of MDS and is a manifestation of disease progression. The time to progress to AML was calculated from the day of randomization to the first day when AML was diagnosed. Participants who died without AML were censored at the date of death. The participants who were lost to follow-up were censored at the last known day when participants did not have AML. Participants who did not progress to AML at the last follow-up contact were censored at the day of the last follow-up contact.
Kaplan Meier Estimate for Overall Survival (OS) From randomization to final data cut-off date of 03 July 2018; maximum survival follow up was 6.4 years Overall survival was assessed using the time between randomization and the date of death or date of censoring. Participants who were alive at a data cutoff date and participants who were lost to follow-up were censored at the last date when participants were known to be alive.
Compliance Rates Using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) From Baseline to Week 48 Baseline, Week 12, (±3 days), Week 24, (±3 days), Week 36, (±3 days), and Week 48 (±3 days); up to data cut-off of 17 Mar 2014 The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). Subscale scores are transformed to a 0 to 100 scale, with higher scores on functional scales indicating better function and higher score on symptom scales indicating worse symptoms. A participant was considered compliant at a visit if at least 15 out of the QLQ-C30 items in the questionnaire were checked.
Mean Change From Baseline in the EORTC QLQ-C30 Dyspnea Domain at Week 12 and 24 Baseline and Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom).
Mean Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain at Week 12 and 24 Baseline and Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Physical Functioning Scale was scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Mean Change From Baseline in the EORTC QLQ-C30 Emotional Functioning Domain at Week 12 and 24 Baseline and Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Emotional Functioning Domain was scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Mean Change From Baseline in the Dyspnea Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24 Baseline, Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom).
Mean Change From Baseline in the EORTC QLQ-C30 Fatigue Domain at Week 12 and 24 Baseline and Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).
Percentage of Participants With a Clinically Meaningful Improvement in QOL (EORTC QLQ-C-30 Scale) From Baseline in Fatigue Domain at Weeks 12 and 24 Baseline, Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). Subscale scores are transformed to a 0 to 100 scale, with higher scores on functional scales indicating better function and higher score on symptom scales indicating worse symptoms. Improvement means at least 10 points better compared to baseline
Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline in the Dyspnea Domain at Weeks 12 and 24 Baseline, Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom). Improvement means at least 10 points better compared to baseline.
Mean Change From Baseline in the EORTC QLQ-C30 Global Health Status/Quality of Life (QOL) Domain at Week 12 and 24 Baseline and Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status/QOL scale was scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.
Mean Change From Baseline in Fatigue Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24 Baseline, Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).
Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline in the Emotional Functioning Domain at Weeks 12 and 24 Baseline, Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Emotional Functioning Domain was scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Healthcare Resource Utilization (HRU): Number of Days of Hospitalization Due to Adverse Events Per Person-Years From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively. Hospitalizations due to adverse events exclude those for transfusions, elective procedures or protocol-driven procedures. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient
Mean Change From Baseline in the Physical Functioning Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24 Baseline, Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). The EORTC QLQ-C30 Physical Functioning was scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.
Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline in the Global Health Status/QOL Domain at Weeks 12 and 24 Baseline, Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). Subscale scores are transformed to a 0 to 100 scale, with higher scores on functional scales indicating better function and higher score on symptom scales indicating worse symptoms. A change of at least 10 points on the standardized domain scores was required for it to be considered clinically meaningful.
Mean Change From Baseline in the Global Health Status/QoL Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24 Baseline, Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). The EORTC QLQ-C30 Global Health Status/QOL scale was scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.
Mean Change From Baseline in the Emotional Functioning Domain Associated With the EORTC QLQ-C30 Scale at Weeks 12 and 24 Baseline, Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Emotional Functioning Scale is scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline Within the Physical Functioning Domain at Weeks 12 and 24 Baseline, Week 12, ±3 days and Week 24, ±3 days The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). Subscale scores are transformed to a 0 to 100 scale, with higher scores on functional scales indicating better function and higher score on symptom scales indicating worse symptoms. A change of at least 10 points on the standardized domain scores was required for it to be considered clinically meaningful.
Healthcare Resource Utilization (HRU): Rate of Inpatient Hospitalizations Related to Adverse Events Per Person Year From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively. Hospitalizations due to adverse events exclude those for transfusions, elective procedures or protocol-driven procedures. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient.
Healthcare Resource Utilization (HRU): Duration of Hospitalizations Due to Adverse Events From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively. Hospitalizations due to adverse events exclude those for transfusions, elective procedures or protocol-driven procedures. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient.
Trial Locations
- Locations (99)
Hackensack University Medical Center
🇺🇸Hackensack, New Jersey, United States
Sunnybrook Regional Cancer Center
🇨🇦Toronto, Ontario, Canada
Southern Illinois Hematology Oncology
🇺🇸Centralia, Illinois, United States
Wollongong Hospital
🇦🇺Wollongong, New South Wales, Australia
Princess Alexandra Hospital
🇦🇺Woolloongabba, Australia
Wiener Gebietskrankenkasse-Hanusch-Krankenhaus
🇦🇹Wien, Austria
Center Hospitalier Universitaire Ambroise Pare
🇧🇪Mons, Belgium
Tom Baker Cancer Center
🇨🇦Calgary, Alberta, Canada
University of Alberta Hospital
🇨🇦Edmonton, Alberta, Canada
Princess Margaret Hospital and University of Toronto
🇨🇦Toronto, Ontario, Canada
McGill University, Dept. Oncology Clinical Research Program
🇨🇦Montreal, Quebec, Canada
Hopital du Sacre-Coeur de Montreal
🇨🇦Montreal, Quebec, Canada
Fakultni nemocnice Brno
🇨🇿Brno, Czechia
Fakultni nemocnice Olomouc
🇨🇿Olomouc, Czechia
Vseobecna Fakultni Nemocnice v Praze
🇨🇿Prague, Czechia
Hopital Avicenne
🇫🇷Bobigny Cedex, France
Ustav hematologie a krevni transfuze
🇨🇿Praha, Czechia
Hopital A. Michallon
🇫🇷La Tronche, France
Institut Paoli-Calmettes
🇫🇷Marseille cedex, France
Groupe hospitalier Cochin Saint-Vincent de Paul
🇫🇷Paris Cedex, France
BAG Freiberg-Richter, Jacobash, Illmer, Wolf
🇩🇪Dresden, Germany
Sankt Johannes Hospital Duisburg
🇩🇪Duisberg, Germany
Universitätsklinikum Düsseldorf
🇩🇪Düesseldorf, Germany
Medizinische Hochschule Hannover
🇩🇪Hannover, Germany
Klinikum Mannheim der Universitat Heidelberg
🇩🇪Heidelberg, Germany
Universitat zu Koln
🇩🇪Köln, Germany
Universitatsklinikum Mannheim
🇩🇪Mannheim, Germany
TU München - Klinikum rechts der Isar
🇩🇪München, Germany
The Chaim Sheba Medical Center
🇮🇱Tel Hashomer, Israel
Rabin Medical Center
🇮🇱Petach-Tikva, Israel
Tel-Aviv Sourasky Medical Center
🇮🇱Tel-Aviv, Israel
Az. Osp. SS.Antonio e Biagio - SC Ematologia
🇮🇹Alessandria, Italy
A.O.U. di Bologna Policlinico S.Orsola-Malpighi
🇮🇹Bologna, Italy
Azienda Ospedaliera Cardarelli
🇮🇹Naples, Italy
P.O. Ospedale Roberto Binaghi (UNI CA/ASL 8)
🇮🇹Cagliari, Italy
Azienda Ospedaliera Universitaria Policlinico Tor Vergata
🇮🇹Roma, Italy
Policlinico Agostino Gemelli - Istituto di Ematologia
🇮🇹Roma, Italy
Tokai University School of Medicine
🇯🇵Isehara City, Kanagawa, Japan
Kameda General Hospital
🇯🇵Kamogawa, Japan
Kanazawa University Hospital
🇯🇵Kanazawa, Japan
Nagasaki Unversity Hospital
🇯🇵Nagasaki, Japan
National Hospital Organization Nagoya Medical Center
🇯🇵Nagoya, Japan
Japanese Red Cross Medical Center
🇯🇵Shibuya, Japan
Instytut Hematologii i Transfuzjologii, Klinika Hematologii
🇵🇱Warsaw, Poland
Hospital Clinic Provincial de Barcelona
🇪🇸Barcelona, Spain
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Dokuz Eylul Universitesi Tip Faiultesi
🇹🇷Izimir, Turkey
Hospital Son Llatzer
🇪🇸Palma de Mallorca, Spain
Hospital Universitario Virgen Del Rocio
🇪🇸Sevilla, Spain
Hospital Universitario de Salamanca
🇪🇸Salamanca, Spain
Hospital Universitario La Fe
🇪🇸Valencia, Spain
Gazi Universitesi Tip Fakltesi
🇹🇷Ankara, Turkey
Istanbul Universitesi Istanbul
🇹🇷Istanbul, Turkey
Royal Bournemouth Hospital
🇬🇧Bournemouth, United Kingdom
University Hospital of Wales
🇬🇧Cardiff, United Kingdom
Saint James University Hospital
🇬🇧Leeds, United Kingdom
Barts Cancer Institute, Queen Mary University of London, Charterhouse Square
🇬🇧London, United Kingdom
Christie Hospital
🇬🇧Manchester, United Kingdom
John Radcliffe Hospital
🇬🇧Oxford, United Kingdom
University of California at Los Angeles
🇺🇸Los Angeles, California, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Cancer Care Manitoba
🇨🇦Winnepeg, Manitoba, Canada
Tohoku University Hospital
🇯🇵Sendai, Japan
Instituto Portugues de Oncologia de Lisboa
🇵🇹Lisboa, Portugal
Akdeniz Universitesi Tip Fakultesi
🇹🇷Antalya, Turkey
University of Texas MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
King's College Hospital
🇬🇧London, United Kingdom
Hospital Universitario Virgen de la Victoria
🇪🇸Malaga, Spain
Grand Hopital de Charleroi
🇧🇪Charleroi, Belgium
Marien Hospital
🇩🇪Duesseldorf, Germany
Hiroshima University Hospital
🇯🇵Hiroshima, Japan
Royal Adelaide Hospital Institute of Medical and Veterinary Science
🇦🇺Adelaide, South Australia, Australia
Medizinische Universitat Innsbruck
🇦🇹Innsbruck, Austria
Universitatsklinik fur Innere Medizin Salzburg
🇦🇹Salzburg, Austria
Cliniques Universitaires UCL de Mont-Godine
🇧🇪Namur, Belgium
Cliniques Universitaires Saint-Luc
🇧🇪Brussels, Belgium
CHU d'Angers
🇫🇷Angers, France
CHRU de Lille-Hopital Claude Huriez Service des Maladies du Sang
🇫🇷Lille, France
Azienda Ospedaliero-Universitaria di Cagliari
🇮🇹Cagliari, Italy
Azienda Ospedaliero-Universitaria Careggi
🇮🇹Firenze, Italy
Osaka Red Cross Hospital
🇯🇵Osaka, Japan
Katedra i Klinika Hematologii i Transplantacji Szpiku - SLASKIEGO UNIWERSYTETU MEDYCZNEGO
🇵🇱Gdansk, Poland
Krankenhaus der Elisabethinen Linz, I Interne Abteilung
🇦🇹Linz, Austria
AZ St-Jan Brugge Oostende AV
🇧🇪Brugge, Belgium
Universitair Ziekenhuis Antwerpen
🇧🇪Edegem, Belgium
Maisonneuve Rosemont
🇨🇦Montreal, Quebec, Canada
AOU San Luigi Gonzaga
🇮🇹Orbassano, Italy
Azienda Policlinico Umberto I, Universita La Sapienzadi Roma
🇮🇹Roma, Italy
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Klinikum Wels-Grieskirchen GmbH
🇦🇹Weis, Austria
Centre Hospitalier Universitaire de Liege
🇧🇪Liege, Belgium
Policlinico Univeristario di Udine
🇮🇹Udine, Italy
Uniwersytet Medyczny w Lodzi
🇵🇱Lodz, Poland
Dartmouth Hitchcock Medical Center Norris Cotton Cancer Center
🇺🇸Lebanon, New Hampshire, United States
IRCCS Centro di Riferimento Oncologico di Basilicata di Rionero in Vulture
🇮🇹Rionero in Vulture, Italy
Jichi Medical University Hospital
🇯🇵Shimotsuke, Japan
Kanto Medical Center NTT EC
🇯🇵Shinagawa, Japan
Hospitais da Universidade de Coimbra
🇵🇹Coimbra, Portugal
Hospital Geral de Santo Antonio
🇵🇹Porto, Portugal