An observational study in Renal Cell Carcinoma, sponsored by Pfizer. Completed at 19 sites in Turkey. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-07-26.
Sponsored by Pfizer · Observational
Metastatic renal cell carcinoma (mRCC) is the most common malignant tumour of the kidneys. Targeted therapies, which were recently introduced in the treatment of mRCC, have become the standard treatment in these patients. With improved survival rate and a tolerable side effect profile, Tyrosine Kinase Inhibitors (TKIs) have largely replaced conventional immunotherapies worldwide.
In Turkey, due to reimbursement conditions, cytokine (interferon alpha) treatment is the standard treatment as first-line therapy.
Therefore, the data on quality of life (QoL) from the pivotal studies with standard TKI treatment does not reflect the QoL status of patients treated with TKIs as second or third line treatment in Turkey. In this study, the clinical outcomes and the impact on quality of life of targeted treatments following TKIs will be explored. To our knowledge, since there is no similar reimbursement condition in the world placing IFN as the first line standard treatment, this will be the first study evaluating the QoL status with targeted therapies used as 3rd line treatment in mRCC patients.
Background:
Metastatic renal cell carcinoma (mRCC) is the most common malignant tumour of the kidneys. Targeted therapies, which were recently introduced in the treatment of mRCC, have become the standard treatment in these patients [1]. With improved survival rate and a tolerable side effect profile, Tyrosine Kinase Inhibitors (TKIs) have largely replaced conventional immunotherapies worldwide. [2-3] In Turkey, due to reimbursement conditions defined by the authority, cytokine (interferon alpha) treatment is the standard treatment as first-line therapy [4].
Rationale:
TKI therapy is used after interferon alpha in Turkey due to current reimbursement status. Therefore, the data on quality of life (QoL) from the pivotal studies with standard TKI treatment does not reflect the QoL status of patients treated with TKIs as second or third line treatment in Turkey. In this study, the clinical outcomes and the impact on quality of life of targeted treatments following TKIs will be explored. To our knowledge, since there is no similar reimbursement condition in the world placing IFN as the first line standard treatment, this will be the first study evaluating the QoL status with targeted therapies used as 3rd line treatment in mRCC patients.
Research Question and Objectives
Research Question:
There is no data available in the literature explaining the effect of targeted therapies used as 3rd line treatment following IFN and TKIs in metastatic renal cell carcinoma patients. Therefore, it is essential to understand health-related quality of life and efficacy of targeted therapies used as 3rd line treatment due to current reimbursement conditions in Turkey.
Primary Objective:
Secondary Objectives:
Study Population:
Metastatic renal cell carcinoma patients under 3rd line targeted therapy following 1st line interferon alpha and 2nd line TKI. All patients will be ≥ 18 years old and have signed the informed consent document.
Variables:
Age, gender, height, weight, and vital signs such as blood pressure Socio-demographic characteristics ECOG performance status Concomitant diseases and medication Medical history Histopathological findings Type of the surgical procedure Treatment history and current treatment information Metastatic features MSKCC risk factors Laboratory findings The quality of life questionnaire (FKSI-15, EQ5D-3L, FKSI-DRS) on each visit. Blood pressure diary Other treatment during follow-up, if any, the dose and the duration Side effects (treatment of the side effects, interruption of the treatment, dose reduction, dose-elevation) PFS, OS and ORR evaluation during 3rd, 6th, 9th and 12th months performed according to RECIST version 1.1, additionally, every patient will be followed-up once for survival follow-up in order to assess the overall survival before the site close out visit. Survival follow-up will be performed via telephone visit or site visit if exist.
ORR: The best overall response is the best response recorded from the start of the study treatment until the end of treatment taking into account any requirement for confirmation. The patient's best overall response assignment will depend on the findings of both target and non-target disease and will also take into consideration the appearance of new lesions. Furthermore, depending on the nature of the study and the protocol requirements, it may also require confirmatory measurement.
PFS: The length of time during and after the treatment of a disease, that a patient lives with the disease but it does not get worse. In a clinical trial, measuring the progression-free survival is one way to see how well a new treatment works.
OS: 1 year Overall Survival (OS), defined as the time from the start of 3rd line treatment until death or 1 year due to any cause (measured at the end of follow-up).
Data Sources: The source of the data will be the electronic or the written patient records of the participating centers. The patient's data can be accessed through a patient file. In the centers, both systems can be used in conjunction. The data can be accessed using both systems.
Sample Size: This is a multi-center, national, non-interventional/observational study. In this study, patients with metastatic renal cell carcinoma who meet the inclusion criteria will be included from centers in 12 NUTS regions of Turkey. Since this is a non-interventional observational study, there is no specific follow-up protocol. In this study, approximately 152 patients will be evaluated.
According to Turkish Statistical Institute data at the end of 2013 population of Turkey is approximately 80 million [5]. In the project of Turkey Association of Cancer Research Control which was named Cancer Record and Incidence shows that cancer incidence is 100-150 in 100.000 [6] and 2% of all new cancers are renal cell carcinoma [7]. There would be 1800 new RCC patients in 1 year, and according to the OS of the disease, there would be 4000 RCC patients in Turkey. 15% of these are metastatic at the time of diagnosis and 30-40% of these are metastatic after a period of a time [8]. Presuming that there are 1800 patients with metastatic renal carcinoma in Turkey, the minimum sample size with 7,6 confidence interval, 95% confidence level and 80% power was calculated as 152.
Data Analysis:
Statistical analyses will be primarily of explorative and descriptive nature. Patients who received at least one dose of 3rd line therapy and have sufficient information whether they had an adverse event or not will be valid for safety analysis. Patients who received at least one dose of 3rd line therapy and have any information regarding efficacy of therapy will be valid for intent-to-treat efficacy analysis.
Demographic data, baseline characteristics, diagnosis and prior treatment of RCC, concomitant diseases, and concomitant medication will be described with summary statistics such as mean, SD, minimum, 1, 5, 25, 75, 95, 99 percent quartiles, median, maximum for continuous variables, and category counts and frequencies (percentages) for categorical variables. Concomitant diseases on the case report form correspond to MedDRA terms. Concomitant medication will be coded using WHO's drug dictionary.
Descriptive summaries of Kaplan-Meier (KM) estimates (including number of failed, number censored, 25th and 75th percentiles with respective 95% confidence level and median with 95%Confidence level) and KM curves will be presented for time-to-event efficacy variables (PFS, TTP, time to treatment failure). Mean, SD, minimum, 1, 5, 25, 75, 95, 99 percent quartiles, median, maximum will be produced for duration of treatment. Category counts and frequencies (percentages) will be calculated for tumor status at different visits and general subjective rating of efficacy of 3rd line therapy from the treating physician.
Adverse events will be summarized using the CTCAE.4.03 coding system. Event rates for single adverse events will be calculated based on the total number of patients valid for safety. Adverse events will be categorized according to relation, seriousness, CTCAE grade (version 4.03), and discontinuation of therapy, action taken and outcome. Special attention will be paid to serious adverse events and unexpected or unlisted ADRs.
Category counts and frequencies (percentages) will be calculated for overall tolerability.
Since the enrollment is after the initiation of the treatment, some information will be collected and analyzed retrospectively.
The best overall response is the best response recorded from the start of the study treatment until the end of treatment taking into account any requirement for confirmation.
6,741 studies on the registry are indexed under Carcinoma; 1,161 are open to participants now.
This study's enrollment of 102 is below the median of 149 across 1,175 observational studies indexed under Carcinoma.
Browse Carcinoma studies →Pfizer is the lead sponsor of 3,244 studies on the registry; 139 are open to participants now.
Of its 582 completed or terminated interventional studies of FDA-regulated products, 381 (65%) have results posted.
Counted across the registry records on this site, refreshed daily.
Metastatic renal cell carcinoma patients under 3rd line targeted therapy following 1st line interferon alpha and 2nd line TKI. All patients will be ≥ 18 years old and have signed the informed consent document.
Patients must meet all of the following inclusion criteria to be eligible for inclusion in the study:
Exclusion criteria:
Patients meeting any of the following criteria will not be included in the study:
Metastatic renal cell carcinoma patients under 3rd line targeted therapy following 1st line interferon alpha and 2nd line TKI. All patients will be ≥ 18 years old and have signed the informed consent document.
Progression Free Survival (PFS)
PFS was defined as the time duration (in months) during and after the treatment of disease that a participant lived with the disease but it did not get worse or progressed. Progressive disease as per response evaluation criteria in solid tumors (RECIST) version 1.1 defined as at least a 20 percent (%) increase in the sum of longest dimensions of target lesions, reference to the smallest sum of the longest dimensions recorded since the treatment started, or the appearance of one or more new lesions or increase of at least 5 millimeter (mm) in addition to the relative increase of 20%.
Time frame: From the start of disease treatment until disease progression or death due to any cause (up to a maximum of 33 months)
Percentage of Participants With Overall Objective Response
Overall objective response was defined as the percentage of participants with best confirmed response (partial response \[PR\], stable disease \[SD\] or progressive disease \[PD\]) recorded from the start of the study treatment until the end of treatment as assessed by RECIST version 1.1. PR defined as a 30% or more decrease in the sum of longest dimensions of the target lesions, taking as reference the baseline sum of longest dimensions. PD defined as at least a 20% increase in the sum of longest dimensions of target lesions, reference to the smallest sum of the longest dimensions recorded since the treatment started, or the appearance of one or more new lesions or increase of at least 5 mm in addition to the relative increase of 20%. SD defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, reference to the smallest sum diameters while on study.
Time frame: Baseline until the maximum of 33 months
Functional Assessment of Cancer Therapy Kidney Symptom Index - 15 (FKSI-15) Score at Baseline
FKSI was used to assess quality of life (QoL) of the participants and consisted of 15 items (lack of energy, side effects, pain, losing weight, bone pain, fatigue, enjoying life, short of breath, worsened condition, appetite, coughing, bothered by fevers, ability to work, hematuria and sleep). Each of the 15 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI score = sum of the 15 item scores; total range: 0 - 60; 0 (no symptoms) to 60 (very much); higher score indicated greater presence of symptoms/worse quality of life.
Time frame: Baseline (Day 1 of Month 1)
Change From Baseline in FKSI-15 Score at Month 3
FKSI was used to assess QoL of the participants and consisted of 15 items (lack of energy, side effects, pain, losing weight, bone pain, fatigue, enjoying life, short of breath, worsened condition, appetite, coughing, bothered by fevers, ability to work, hematuria and sleep). Each of the 15 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI score = sum of the 15 item scores; total range: 0 - 60; 0 (no symptoms) to 60 (very much); higher score indicated greater presence of symptoms/worse quality of life.
Time frame: Baseline (Day 1 of Month 1), Month 3
Change From Baseline in FKSI-15 Score at Month 6
FKSI was used to assess quality of life (QoL) of the participants and consisted of 15 items (lack of energy, side effects, pain, losing weight, bone pain, fatigue, enjoying life, short of breath, worsened condition, appetite, coughing, bothered by fevers, ability to work, hematuria and sleep). Each of the 15 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI score = sum of the 15 item scores; total range: 0 - 60; 0 (no symptoms) to 60 (very much); higher score indicated greater presence of symptoms/worse quality of life.
Time frame: Baseline (Day 1 of Month 1), Month 6
Change From Baseline in FKSI-15 Score at Month 9
FKSI was used to assess quality of life (QoL) of the participants and consisted of 15 items (lack of energy, side effects, pain, losing weight, bone pain, fatigue, enjoying life, short of breath, worsened condition, appetite, coughing, bothered by fevers, ability to work, hematuria and sleep). Each of the 15 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI score = sum of the 15 item scores; total range: 0 - 60; 0 (no symptoms) to 60 (very much); higher score indicated greater presence of symptoms/worse quality of life.
Time frame: Baseline (Day 1 of Month 1), Month 9
Change From Baseline in FKSI-15 Score at Last Follow-up
FKSI was used to assess quality of life (QoL) of the participants and consisted of 15 items (lack of energy, side effects, pain, losing weight, bone pain, fatigue, enjoying life, short of breath, worsened condition, appetite, coughing, bothered by fevers, ability to work, hematuria and sleep). Each of the 15 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI score = sum of the 15 item scores; total range: 0 - 60; 0 (no symptoms) to 60 (very much); higher score indicated greater presence of symptoms/worse quality of life.
Time frame: Baseline (Day 1 of Month 1), last follow-up visit (up to 33 months)
EuroQol-5 Dimension-3 Level (EQ5D-3L) Scores at Baseline
EQ-5D-3L, a health profile questionnaire was used to assess quality of life along 5 dimensions. Participants rated 5 aspects of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) by choosing from 3 answering options (1=no problems; 2=some problems; 3=extreme problems). The mean of the summed score ranged from 1 to 3 with "1" corresponding to no problems and "3" corresponding to severe problems in the 5 dimensions, where higher score indicates more severe problems. Total index EQ-5D-3L summary score is weighted with a range of -0.594 (worst) to 1.0 (best).
Time frame: Baseline (Day 1 of Month 1)
Change From Baseline in EQ5D-3L Scores at Month 3
EQ-5D-3L, a health profile questionnaire was used to assess quality of life along 5 dimensions. Participants rated 5 aspects of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) by choosing from 3 answering options (1=no problems; 2=some problems; 3=extreme problems). The summed score ranged from 1-15 with "1" corresponding to no problems and "15" corresponding to severe problems in the 5 dimensions, where higher score indicates more severe problems.
Time frame: Baseline (Day 1 of Month 1), Month 3
Change From Baseline in EQ5D-3L Score at Month 6
EQ-5D-3L, a health profile questionnaire was used to assess quality of life along 5 dimensions. Participants rated 5 aspects of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) by choosing from 3 answering options (1=no problems; 2=some problems; 3=extreme problems). The summed score ranged from 1-15 with "1" corresponding to no problems and "15" corresponding to severe problems in the 5 dimensions, where higher score indicates more severe problems.
Time frame: Baseline (Day 1 of Month 1), Month 6
Change From Baseline in EQ5D-3L Score at Month 9
EQ-5D-3L, a health profile questionnaire was used to assess quality of life along 5 dimensions. Participants rated 5 aspects of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) by choosing from 3 answering options (1=no problems; 2=some problems; 3=extreme problems). The summed score ranged from 1-15 with "1" corresponding to no problems and "15" corresponding to severe problems in the 5 dimensions, where higher score indicates more severe problems.
Time frame: Baseline (Day 1 of Month 1), Month 9
Change From Baseline in EQ5D-3L Score at Last Follow-up
EQ-5D-3L, a health profile questionnaire was used to assess quality of life along 5 dimensions. Participants rated 5 aspects of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) by choosing from 3 answering options (1=no problems; 2=some problems; 3=extreme problems). The summed score ranged from 1-15 with "1" corresponding to no problems and "15" corresponding to severe problems in the 5 dimensions, where higher score indicates more severe problems.
Time frame: Baseline (Day 1 of Month 1), last follow up visit (up to 33 months)
Functional Assessment of Cancer Therapy Kidney Symptom Index-Disease-Related Symptoms (FKSI-DRS) Score at Baseline
FKSI-DRS was used to assess quality of life in participants and consisted of 9 items (lack of energy, pain, losing weight, bone pain, fatigue, short of breath, coughing, bothered by fevers, and hematuria). Each of the 9 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI-DRS score = sum of the 9 item scores; total range: 0 - 36; 0 (no symptom) to 36 (very much); higher score indicated greater presence of symptom.
Time frame: Baseline (Day 1 of Month 1)
Change From Baseline in FKSI-DRS Score at Month 3
FKSI-DRS was used to assess quality of life in participants and consisted of 9 items (lack of energy, pain, losing weight, bone pain, fatigue, short of breath, coughing, bothered by fevers, and hematuria). Each of the 9 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI-DRS score = sum of the 9 item scores; total range: 0 - 36; 0 (no symptom) to 36 (very much); higher score indicated greater presence of symptom.
Time frame: Baseline (Day 1 of Month 1), Month 3
Change From Baseline in FKSI-DRS Score at Month 6
FKSI-DRS was used to assess quality of life in participants and consisted of 9 items (lack of energy, pain, losing weight, bone pain, fatigue, short of breath, coughing, bothered by fevers, and hematuria). Each of the 9 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI-DRS score = sum of the 9 item scores; total range: 0 - 36; 0 (no symptom) to 36 (very much); higher score indicated greater presence of symptom.
Time frame: Baseline (Day 1 of Month 1), Month 6
Change From Baseline in FKSI-DRS Score at Month 9
FKSI-DRS was used to assess quality of life in participants and consisted of 9 items (lack of energy, pain, losing weight, bone pain, fatigue, short of breath, coughing, bothered by fevers, and hematuria). Each of the 9 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI-DRS score = sum of the 9 item scores; total range: 0 - 36; 0 (no symptom) to 36 (very much); higher score indicated greater presence of symptom.
Time frame: Baseline (Day 1 of Month 1), Month 9
Change From Baseline in FKSI-DRS Score at Last Follow-up
FKSI-DRS was used to assess quality of life in participants and consisted of 9 items (lack of energy, pain, losing weight, bone pain, fatigue, short of breath, coughing, bothered by fevers, and hematuria). Each of the 9 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI-DRS score = sum of the 9 item scores; total range: 0 - 36; 0 (no symptom) to 36 (very much); higher score indicated greater presence of symptom.
Time frame: Baseline (Day 1 of Month 1), last follow up visit (up to 33 months)
Overall Survival
Overall survival was defined as the time from the start of 3rd line treatment until date of death due to any cause.
Time frame: From the start of 3rd line treatment until death due to any cause (up to a maximum of 33 months)
Overall Survival at Year 1: Percentage of Participants Who Survived at Year 1
Overall survival at Year 1 (Month 12) was defined as the time from the start of 3rd line treatment until death or 1 year due to any cause (measured at the end of 12 month follow-up/observation period). Percentage of participants who survived at the completion of 1 year (12 months) period were reported in this outcome measure.
Time frame: Baseline until death due to any cause (up to 1 year)
Time to Treatment Failure
Time to treatment failure was defined as the time from the start of treatment to the date of disease progression or date of permanent discontinuation. PD as per RECIST version 1.1 was defined as at least a 20% increase in the sum of longest dimensions of target lesions, reference to the smallest sum of the longest dimensions recorded since the treatment started, or the appearance of one or more new lesions or increase of at least 5 mm in addition to the relative increase of 20%.
Time frame: Baseline until disease progression or discontinuation, due to any cause (up to 33 months)
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE was any untoward medical occurrence in a participant who received disease treatment without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant and jeopardized the participants or required treatment to prevent other AE outcomes for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment emergent AEs were events which occurred between start of disease treatment and up to Month 12 follow-up visit, that were absent before treatment or that worsened relative to pre-treatment state. AEs included both non-SAE and SAEs.
Time frame: Baseline up to 12 months
Number of Participants With Grade 3 or Higher Severe Adverse Events (AEs) Based on NCI CTCAE Version 4.03
An AE was any untoward medical occurrence in a participant who received disease treatment without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant and jeopardized the participants or required treatment to prevent other AE outcomes for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs were graded according to the National Cancer Institute- Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.03 and coded using the Medical Dictionary for Regulatory Activities (MedDRA) as Grade 1: Mild, Grade 2: Moderate, Grade 3: Severe, Grade 4: Life threatening, Grade 5: Death related to AE. AEs included both SAEs and non-SAEs.
Time frame: Baseline up to 12 months
Number of Participants With Treatment-Emergent Adverse Events (AEs) During Third Line Targeted Treatment
An AE was any untoward medical occurrence in a participant who received disease treatment without regard to possibility of causal relationship. Treatment emergent AEs during third line targeted treatment were events which occurred between first dose of third line targeted treatment and up to Month 12 follow-up visit, that were absent before treatment or that worsened relative to pre-treatment state.
Time frame: Baseline up to 12 months
Number of Participants With Dose Modifications of Third-Line Treatment Due to Adverse Events
Number of participants that required dose modifications of third line treatment due to AEs were reported in this outcome measure. Dose modification was categorized as escalation (increase in dose), delay or interruptions (change in dose time or skipping any dose), reduction (decrease in dose).
Time frame: Baseline up to Month 3, 6, 9 and 12
Correlation Coefficient Between Efficacy and Dose Modifications Due to AEs
Time frame: Baseline up to Month 12 follow-up visit
Correlation Coefficient Between Efficacy and High Blood Pressure (>150 / 90 Millimeter of Mercury )
Time frame: Baseline up to Month 12 follow-up visit
| Milestone | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Started | 102 |
| Completed | 102 |
| Not completed | 0 |
PFS was defined as the time duration (in months) during and after the treatment of disease that a participant lived with the disease but it did not get worse or progressed. Progressive disease as per response evaluation criteria in solid tumors (RECIST) version 1.1 defined as at least a 20 percent (%) increase in the sum of longest dimensions of target lesions, reference to the smallest sum of the longest dimensions recorded since the treatment started, or the appearance of one or more new lesions or increase of at least 5 millimeter (mm) in addition to the relative increase of 20%.
| months | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Progression Free Survival (PFS) | NA (NA to NA) |
Overall objective response was defined as the percentage of participants with best confirmed response (partial response \[PR\], stable disease \[SD\] or progressive disease \[PD\]) recorded from the start of the study treatment until the end of treatment as assessed by RECIST version 1.1. PR defined as a 30% or more decrease in the sum of longest dimensions of the target lesions, taking as reference the baseline sum of longest dimensions. PD defined as at least a 20% increase in the sum of longest dimensions of target lesions, reference to the smallest sum of the longest dimensions recorded since the treatment started, or the appearance of one or more new lesions or increase of at least 5 mm in addition to the relative increase of 20%. SD defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, reference to the smallest sum diameters while on study.
| percentage of participants | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| PR | 23.5 |
| SD | 38.2 |
| PD | 18.6 |
FKSI was used to assess quality of life (QoL) of the participants and consisted of 15 items (lack of energy, side effects, pain, losing weight, bone pain, fatigue, enjoying life, short of breath, worsened condition, appetite, coughing, bothered by fevers, ability to work, hematuria and sleep). Each of the 15 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI score = sum of the 15 item scores; total range: 0 - 60; 0 (no symptoms) to 60 (very much); higher score indicated greater presence of symptoms/worse quality of life.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Functional Assessment of Cancer Therapy Kidney Symptom Index - 15 (FKSI-15) Score at Baseline | 26 ± 6 |
FKSI was used to assess QoL of the participants and consisted of 15 items (lack of energy, side effects, pain, losing weight, bone pain, fatigue, enjoying life, short of breath, worsened condition, appetite, coughing, bothered by fevers, ability to work, hematuria and sleep). Each of the 15 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI score = sum of the 15 item scores; total range: 0 - 60; 0 (no symptoms) to 60 (very much); higher score indicated greater presence of symptoms/worse quality of life.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in FKSI-15 Score at Month 3 | 1.2 ± 4.47 |
FKSI was used to assess quality of life (QoL) of the participants and consisted of 15 items (lack of energy, side effects, pain, losing weight, bone pain, fatigue, enjoying life, short of breath, worsened condition, appetite, coughing, bothered by fevers, ability to work, hematuria and sleep). Each of the 15 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI score = sum of the 15 item scores; total range: 0 - 60; 0 (no symptoms) to 60 (very much); higher score indicated greater presence of symptoms/worse quality of life.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in FKSI-15 Score at Month 6 | 1.21 ± 4.22 |
FKSI was used to assess quality of life (QoL) of the participants and consisted of 15 items (lack of energy, side effects, pain, losing weight, bone pain, fatigue, enjoying life, short of breath, worsened condition, appetite, coughing, bothered by fevers, ability to work, hematuria and sleep). Each of the 15 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI score = sum of the 15 item scores; total range: 0 - 60; 0 (no symptoms) to 60 (very much); higher score indicated greater presence of symptoms/worse quality of life.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in FKSI-15 Score at Month 9 | 1.38 ± 4.56 |
FKSI was used to assess quality of life (QoL) of the participants and consisted of 15 items (lack of energy, side effects, pain, losing weight, bone pain, fatigue, enjoying life, short of breath, worsened condition, appetite, coughing, bothered by fevers, ability to work, hematuria and sleep). Each of the 15 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI score = sum of the 15 item scores; total range: 0 - 60; 0 (no symptoms) to 60 (very much); higher score indicated greater presence of symptoms/worse quality of life.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in FKSI-15 Score at Last Follow-up | 0.67 ± 5.27 |
EQ-5D-3L, a health profile questionnaire was used to assess quality of life along 5 dimensions. Participants rated 5 aspects of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) by choosing from 3 answering options (1=no problems; 2=some problems; 3=extreme problems). The mean of the summed score ranged from 1 to 3 with "1" corresponding to no problems and "3" corresponding to severe problems in the 5 dimensions, where higher score indicates more severe problems. Total index EQ-5D-3L summary score is weighted with a range of -0.594 (worst) to 1.0 (best).
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| EuroQol-5 Dimension-3 Level (EQ5D-3L) Scores at Baseline | 0.522 ± 0.236 |
EQ-5D-3L, a health profile questionnaire was used to assess quality of life along 5 dimensions. Participants rated 5 aspects of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) by choosing from 3 answering options (1=no problems; 2=some problems; 3=extreme problems). The summed score ranged from 1-15 with "1" corresponding to no problems and "15" corresponding to severe problems in the 5 dimensions, where higher score indicates more severe problems.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in EQ5D-3L Scores at Month 3 | -0.07 ± 0.17 |
EQ-5D-3L, a health profile questionnaire was used to assess quality of life along 5 dimensions. Participants rated 5 aspects of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) by choosing from 3 answering options (1=no problems; 2=some problems; 3=extreme problems). The summed score ranged from 1-15 with "1" corresponding to no problems and "15" corresponding to severe problems in the 5 dimensions, where higher score indicates more severe problems.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in EQ5D-3L Score at Month 6 | -0.11 ± 0.34 |
EQ-5D-3L, a health profile questionnaire was used to assess quality of life along 5 dimensions. Participants rated 5 aspects of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) by choosing from 3 answering options (1=no problems; 2=some problems; 3=extreme problems). The summed score ranged from 1-15 with "1" corresponding to no problems and "15" corresponding to severe problems in the 5 dimensions, where higher score indicates more severe problems.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in EQ5D-3L Score at Month 9 | -0.06 ± 0.2 |
EQ-5D-3L, a health profile questionnaire was used to assess quality of life along 5 dimensions. Participants rated 5 aspects of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) by choosing from 3 answering options (1=no problems; 2=some problems; 3=extreme problems). The summed score ranged from 1-15 with "1" corresponding to no problems and "15" corresponding to severe problems in the 5 dimensions, where higher score indicates more severe problems.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in EQ5D-3L Score at Last Follow-up | -0.05 ± 0.39 |
FKSI-DRS was used to assess quality of life in participants and consisted of 9 items (lack of energy, pain, losing weight, bone pain, fatigue, short of breath, coughing, bothered by fevers, and hematuria). Each of the 9 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI-DRS score = sum of the 9 item scores; total range: 0 - 36; 0 (no symptom) to 36 (very much); higher score indicated greater presence of symptom.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Functional Assessment of Cancer Therapy Kidney Symptom Index-Disease-Related Symptoms (FKSI-DRS) Score at Baseline | 14.56 ± 5.73 |
FKSI-DRS was used to assess quality of life in participants and consisted of 9 items (lack of energy, pain, losing weight, bone pain, fatigue, short of breath, coughing, bothered by fevers, and hematuria). Each of the 9 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI-DRS score = sum of the 9 item scores; total range: 0 - 36; 0 (no symptom) to 36 (very much); higher score indicated greater presence of symptom.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in FKSI-DRS Score at Month 3 | 1.13 ± 3.81 |
FKSI-DRS was used to assess quality of life in participants and consisted of 9 items (lack of energy, pain, losing weight, bone pain, fatigue, short of breath, coughing, bothered by fevers, and hematuria). Each of the 9 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI-DRS score = sum of the 9 item scores; total range: 0 - 36; 0 (no symptom) to 36 (very much); higher score indicated greater presence of symptom.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in FKSI-DRS Score at Month 6 | 1.04 ± 4.98 |
FKSI-DRS was used to assess quality of life in participants and consisted of 9 items (lack of energy, pain, losing weight, bone pain, fatigue, short of breath, coughing, bothered by fevers, and hematuria). Each of the 9 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI-DRS score = sum of the 9 item scores; total range: 0 - 36; 0 (no symptom) to 36 (very much); higher score indicated greater presence of symptom.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in FKSI-DRS Score at Month 9 | 0.68 ± 4.94 |
FKSI-DRS was used to assess quality of life in participants and consisted of 9 items (lack of energy, pain, losing weight, bone pain, fatigue, short of breath, coughing, bothered by fevers, and hematuria). Each of the 9 items was answered on a 5-point Likert scale ranging from 0 to 4 (0= not at all, 1= a little bit, 2= somewhat, 3= quite a bit, 4= very much). Total FKSI-DRS score = sum of the 9 item scores; total range: 0 - 36; 0 (no symptom) to 36 (very much); higher score indicated greater presence of symptom.
| units on a scale | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Change From Baseline in FKSI-DRS Score at Last Follow-up | 1.6 ± 6.66 |
Overall survival was defined as the time from the start of 3rd line treatment until date of death due to any cause.
| months | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Overall Survival | NA (NA to NA) |
Overall survival at Year 1 (Month 12) was defined as the time from the start of 3rd line treatment until death or 1 year due to any cause (measured at the end of 12 month follow-up/observation period). Percentage of participants who survived at the completion of 1 year (12 months) period were reported in this outcome measure.
| percentage of participants | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Overall Survival at Year 1: Percentage of Participants Who Survived at Year 1 | 79.9 |
Time to treatment failure was defined as the time from the start of treatment to the date of disease progression or date of permanent discontinuation. PD as per RECIST version 1.1 was defined as at least a 20% increase in the sum of longest dimensions of target lesions, reference to the smallest sum of the longest dimensions recorded since the treatment started, or the appearance of one or more new lesions or increase of at least 5 mm in addition to the relative increase of 20%.
| months | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Time to Treatment Failure | 4 (1 to 16) |
An AE was any untoward medical occurrence in a participant who received disease treatment without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant and jeopardized the participants or required treatment to prevent other AE outcomes for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment emergent AEs were events which occurred between start of disease treatment and up to Month 12 follow-up visit, that were absent before treatment or that worsened relative to pre-treatment state. AEs included both non-SAE and SAEs.
| Participants | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| AEs | 33 |
| SAEs | 5 |
An AE was any untoward medical occurrence in a participant who received disease treatment without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant and jeopardized the participants or required treatment to prevent other AE outcomes for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs were graded according to the National Cancer Institute- Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.03 and coded using the Medical Dictionary for Regulatory Activities (MedDRA) as Grade 1: Mild, Grade 2: Moderate, Grade 3: Severe, Grade 4: Life threatening, Grade 5: Death related to AE. AEs included both SAEs and non-SAEs.
| Participants | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Number of Participants With Grade 3 or Higher Severe Adverse Events (AEs) Based on NCI CTCAE Version 4.03 | 13 |
An AE was any untoward medical occurrence in a participant who received disease treatment without regard to possibility of causal relationship. Treatment emergent AEs during third line targeted treatment were events which occurred between first dose of third line targeted treatment and up to Month 12 follow-up visit, that were absent before treatment or that worsened relative to pre-treatment state.
| Participants | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (AEs) During Third Line Targeted Treatment | 6 |
Number of participants that required dose modifications of third line treatment due to AEs were reported in this outcome measure. Dose modification was categorized as escalation (increase in dose), delay or interruptions (change in dose time or skipping any dose), reduction (decrease in dose).
| Participants | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Escalation: Month 3 | 7 |
| Interruption/delay: Month 3 | 13 |
| Reduction: Month 3 | 4 |
| None: Month 3 | 58 |
| Escalation: Month 6 | 5 |
| Interruption/delay: Month 6 | 7 |
| Reduction: Month 6 | 2 |
| None: Month 6 | 43 |
| Escalation: Month 9 | 0 |
| Interruption/delay: Month 9 | 5 |
| Reduction: Month 9 | 0 |
| None: Month 9 | 33 |
| Escalation: Month 12 | 0 |
| Interruption/delay: Month 12 | 4 |
| Reduction: Month 12 | 0 |
| None: Month 12 | 25 |
| Correlation coefficient | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Correlation Coefficient Between Efficacy and Dose Modifications Due to AEs | NA |
| Correlation coefficient | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Correlation Coefficient Between Efficacy and High Blood Pressure (>150 / 90 Millimeter of Mercury ) | NA |
Collected over Baseline up to 12 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Metastatic Renal Cell Carcinoma (mRCC) Cohort | 35/102 (34.3%) | 5/102 (4.9%) | 33/102 (32.4%) |
| Event | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Gastrointestinal haemorrhageGastrointestinal disorders | 1/102 |
| PneumoniaInfections and infestations | 1/102 |
| Blood creatinine increasedInvestigations | 1/102 |
| MetastasisNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/102 |
| Mass in right on brochisGeneral disorders | 1/102 |
| Event | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| DiarrhoeaGastrointestinal disorders | 8/102 |
| AstheniaGeneral disorders | 8/102 |
| HypertensionVascular disorders | 5/102 |
| CoughRespiratory, thoracic and mediastinal disorders | 3/102 |
| NauseaGastrointestinal disorders | 3/102 |
| Oral painGastrointestinal disorders | 3/102 |
| HypothyroidismEndocrine disorders | 2/102 |
| VomitingGastrointestinal disorders | 2/102 |
| StomatitisGastrointestinal disorders | 2/102 |
| Deep vein thrombosisVascular disorders | 2/102 |
Baseline analysis population included all enrolled participants.
| Age, Continuous(years) | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Mean | 60 ± 12 |
| Sex: Female, Male(Participants) | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|---|
| Female | 28 |
| Male | 74 |
| Race and Ethnicity Not Collected(Participants) | Metastatic Renal Cell Carcinoma (mRCC) Cohort |
|---|
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Plan to share: No — Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
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