Trial Outcomes & Findings for BAY43-9006 - Phase II Study in Non-Small Cell Lung Carcinoma (NSCLC) (NCT NCT00101413)

NCT ID: NCT00101413

Last Updated: 2013-10-30

Results Overview

CR-disappearance of clinical/radiological tumor evidence (target/nontarget). PR- \>=30% decrease in sum longest diameter (LD) of target lesions from BL sum LD. Stable disease (SD)-no shrinkage for PR nor increase for PD. Progressive disease (PD) measurement proven- \>=20% increase in sum LD of lesions from smallest sum LD since start or new lesions. Progression by clinical judgement- \>clinically meaningful cancer-related deterioration as judged by the investigator.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

52 participants

Primary outcome timeframe

First patient first treatment until date for last data collection for efficacy for a study period up to 62 weeks. Tumor assessed per RECIST at baseline (BL), every 8 weeks during treatment and at end of treatment.

Results posted on

2013-10-30

Participant Flow

Study conducted in Germany and USA (1 center each) from Apr 2004 to Jun 2005. Recruitment in 2 phases: Interim analysis (IA) to be performed after treatment of 29 patients; final analysis planned after additional 21 patients. Final analysis data cut-off date 30 Jun 2005; safety data collected for the 4 ongoing subjects until 11 Apr 2008.

52 of 54 enrolled patients started treatment. The analysis population consisted of 52 subjects for the intent to treat (ITT) and safety analyses and 51 subjects in the evaluable subject analysis (1 had lung metastases from pancreatic cancer).

Participant milestones

Participant milestones
Measure
Sorafenib
Sorafenib (Nexavar, BAY43-9006) 400 mg twice daily (bid, bis in die) X 28 day cycles
Overall Study
STARTED
52
Overall Study
COMPLETED
52
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

BAY43-9006 - Phase II Study in Non-Small Cell Lung Carcinoma (NSCLC)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Sorafenib
n=52 Participants
Sorafenib (Nexavar, BAY43-9006) 400 mg twice daily (bid, bis in die) X 28 day cycles
Age Continuous
62 years
n=5 Participants
Age, Customized
>=65 years
21 participants
n=5 Participants
Age, Customized
< 65 years
31 participants
n=5 Participants
Sex: Female, Male
Female
18 Participants
n=5 Participants
Sex: Female, Male
Male
34 Participants
n=5 Participants
Histology
Adenocarcinoma NSCLC
28 participants
n=5 Participants
Histology
Large cell carcinoma
4 participants
n=5 Participants
Histology
Squamous cell (epidermoid) carcinoma
16 participants
n=5 Participants
Histology
Undifferentiated carcinoma
4 participants
n=5 Participants

PRIMARY outcome

Timeframe: First patient first treatment until date for last data collection for efficacy for a study period up to 62 weeks. Tumor assessed per RECIST at baseline (BL), every 8 weeks during treatment and at end of treatment.

Population: The analysis population for the intent to treat (ITT) and safety analyses consisted of 52 subjects who received at least 1 treatment and had their disease re-evaluated. 51 subjects were considered evaluable since 1 of the 52 subjects had lung metastases from pancreatic cancer.

CR-disappearance of clinical/radiological tumor evidence (target/nontarget). PR- \>=30% decrease in sum longest diameter (LD) of target lesions from BL sum LD. Stable disease (SD)-no shrinkage for PR nor increase for PD. Progressive disease (PD) measurement proven- \>=20% increase in sum LD of lesions from smallest sum LD since start or new lesions. Progression by clinical judgement- \>clinically meaningful cancer-related deterioration as judged by the investigator.

Outcome measures

Outcome measures
Measure
Sorafenib
n=51 Participants
Sorafenib (Nexavar, BAY43-9006) 400 mg twice daily (bid, bis in die) X 28 day cycles
Anti-cancer Activity (eg, Percentage of Patients With Confirmed Complete Responses (CR) and Partial Responses (PR) Per RECIST (Response Evaluation Criteria in Solid Tumors) Criteria in Patients With Stage IV Non-small Cell Lung Carcinoma (NSCLC)
Partial response
0.0 percentage of participants
Anti-cancer Activity (eg, Percentage of Patients With Confirmed Complete Responses (CR) and Partial Responses (PR) Per RECIST (Response Evaluation Criteria in Solid Tumors) Criteria in Patients With Stage IV Non-small Cell Lung Carcinoma (NSCLC)
Progression by clinical judgement
11.8 percentage of participants
Anti-cancer Activity (eg, Percentage of Patients With Confirmed Complete Responses (CR) and Partial Responses (PR) Per RECIST (Response Evaluation Criteria in Solid Tumors) Criteria in Patients With Stage IV Non-small Cell Lung Carcinoma (NSCLC)
Not evaluated
5.9 percentage of participants
Anti-cancer Activity (eg, Percentage of Patients With Confirmed Complete Responses (CR) and Partial Responses (PR) Per RECIST (Response Evaluation Criteria in Solid Tumors) Criteria in Patients With Stage IV Non-small Cell Lung Carcinoma (NSCLC)
Stable disease
58.8 percentage of participants
Anti-cancer Activity (eg, Percentage of Patients With Confirmed Complete Responses (CR) and Partial Responses (PR) Per RECIST (Response Evaluation Criteria in Solid Tumors) Criteria in Patients With Stage IV Non-small Cell Lung Carcinoma (NSCLC)
Progressive disease measurement proven
23.5 percentage of participants
Anti-cancer Activity (eg, Percentage of Patients With Confirmed Complete Responses (CR) and Partial Responses (PR) Per RECIST (Response Evaluation Criteria in Solid Tumors) Criteria in Patients With Stage IV Non-small Cell Lung Carcinoma (NSCLC)
Complete response + Partial response
0.0 percentage of participants
Anti-cancer Activity (eg, Percentage of Patients With Confirmed Complete Responses (CR) and Partial Responses (PR) Per RECIST (Response Evaluation Criteria in Solid Tumors) Criteria in Patients With Stage IV Non-small Cell Lung Carcinoma (NSCLC)
Complete response
0.0 percentage of participants

SECONDARY outcome

Timeframe: First patient first treatment until date for last data collection for efficacy for a study period up to 62 weeks. Tumor assessed per RECIST at baseline (BL), every 8 weeks during treatment and at end of treatment.

Population: The analysis population for the intent to treat (ITT) and safety analyses consisted of 52 subjects who received at least 1 treatment and had their disease re-evaluated. 1 of the 52 subjects had lung metastases from pancreatic cancer and was excluded from analysis. 48 subjects had tumor evaluations post-baseline and were evaluable.

Duration of stable disease was calculated as date of first treatment until date of documented progressive disease (PD) or last observation if subject did not progress. Stable disease (SD) defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. Kaplan-Meier methodology, descriptive analysis.

Outcome measures

Outcome measures
Measure
Sorafenib
n=48 Participants
Sorafenib (Nexavar, BAY43-9006) 400 mg twice daily (bid, bis in die) X 28 day cycles
Duration of Stable Disease
103 days
Interval 58.0 to 116.0

SECONDARY outcome

Timeframe: First patient first treatment until date for last data collection for efficacy for a study period up to 62 weeks.

Population: The analysis population for the intent to treat (ITT) and safety analyses consisted of 52 subjects who received at least 1 treatment and had their disease re-evaluated. 51 subjects were considered evaluable since 1 of the 52 subjects had lung metastases from pancreatic cancer.

Overall survival was calculated from the date of the first treatment until death of the subject. Evaluation by Kaplan-Meier methodology, descriptive analysis.

Outcome measures

Outcome measures
Measure
Sorafenib
n=51 Participants
Sorafenib (Nexavar, BAY43-9006) 400 mg twice daily (bid, bis in die) X 28 day cycles
Overall Survival
205 days
Interval 151.0 to 257.0

SECONDARY outcome

Timeframe: First patient first treatment until date for last data collection for efficacy for a study period up to 62 weeks. Tumor assessed per RECIST at baseline (BL), every 8 weeks during treatment and at end of treatment.

Population: The analysis population for the intent to treat (ITT) and safety analyses consisted of 52 subjects who received at least 1 treatment and had their disease re-evaluated. 51 subjects were considered evaluable since 1 of the 52 subjects had lung metastases from pancreatic cancer.

Percentage of subjects with stable disease was calculated from date of first treatment until date of documented progressive disease (PD) or last observation if subject did not progress. Stable disease (SD) defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. Descriptive summary of subjects with SD.

Outcome measures

Outcome measures
Measure
Sorafenib
n=51 Participants
Sorafenib (Nexavar, BAY43-9006) 400 mg twice daily (bid, bis in die) X 28 day cycles
Percentage of Subjects With Stable Disease (SD)
58.8 Percentage of participants

SECONDARY outcome

Timeframe: From first patient first treatment until date of last efficacy data collection (study period up to 62 weeks). HRQoL assessed at baseline (BL), end of treatment Cycles 2 and 4, and at end of treatment

Population: The analysis population for the intent to treat (ITT) and safety analyses consisted of 52 subjects who received at least 1 treatment and had their disease re-evaluated. Of the 52 treated subjects, 50 subjects completed the FACT-L at baseline (screening) and post-treatment.

HRQoL was assessed with the FACT-L questionnaire, a validated instrument for determining lung cancer HRQoL. The 36-item questionnaire includes 4 domains: Physical, functional, emotional, and social/family well-being, and a lung cancer-specific subscale. The FACT-L total score ranges from 1 to 136. Lower scores (negative change from baseline) demonstrate impaired HRQoL.

Outcome measures

Outcome measures
Measure
Sorafenib
n=50 Participants
Sorafenib (Nexavar, BAY43-9006) 400 mg twice daily (bid, bis in die) X 28 day cycles
Change From Baseline of Health-Related Quality of Life (HRQOL) Score Assessed at Cycle 2, Cycle 4, and End of Treatment (EOT)
Cycle 4
0.0 scores on a scale
Standard Deviation 14.4
Change From Baseline of Health-Related Quality of Life (HRQOL) Score Assessed at Cycle 2, Cycle 4, and End of Treatment (EOT)
Cycle 2
-4.8 scores on a scale
Standard Deviation 21.2
Change From Baseline of Health-Related Quality of Life (HRQOL) Score Assessed at Cycle 2, Cycle 4, and End of Treatment (EOT)
End of treatment
-14.9 scores on a scale
Standard Deviation 23.2

Adverse Events

Sorafenib

Serious events: 28 serious events
Other events: 51 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Sorafenib
n=52 participants at risk
Sorafenib (Nexavar, BAY43-9006) 400 mg twice daily (bid, bis in die) X 28 day cycles
Blood and lymphatic system disorders
Hemoglobin
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Blood and lymphatic system disorders
Platelets
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Cardiac disorders
Cardiac arrythmia - other
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Cardiac disorders
Cardiac general - other
11.5%
6/52 • AE data were collected from Apr 2004 to Apr 2008
Cardiac disorders
Hypertension
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Cardiac disorders
Hypotension
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Death not associated with ctcae term, disease progression nos
11.5%
6/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Fatigue
9.6%
5/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Constitutional symptoms - other
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Vomiting
3.8%
2/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Anorexia
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Fistula, GI, esophagus
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
GI - other
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Stricture, GI,
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Vascular disorders
Hemorrhage, GI, upper GI nos
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Vascular disorders
Hemorrhage pulmonary, respiratory tract nos
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Hepatobiliary disorders
Hepatobiliary -other
3.8%
2/52 • AE data were collected from Apr 2004 to Apr 2008
Hepatobiliary disorders
Pancreatitis
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Infections and infestations
Infection (documented clinically), lung pneumonia
3.8%
2/52 • AE data were collected from Apr 2004 to Apr 2008
Blood and lymphatic system disorders
Dermal change
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Blood and lymphatic system disorders
Edema: limb
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Metabolism and nutrition disorders
Bilirubin
3.8%
2/52 • AE data were collected from Apr 2004 to Apr 2008
Metabolism and nutrition disorders
Amylase
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Metabolism and nutrition disorders
Hyperuricemia
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Metabolism and nutrition disorders
Lipase
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Metabolism and nutrition disorders
Metabolic/lab - other
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Nervous system disorders
CNS ischemia
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Nervous system disorders
Confusion
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Nervous system disorders
Mental status
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Nervous system disorders
Neurology - other
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Nervous system disorders
Somnolence
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Pain, abdomen nos
3.8%
2/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, chest/thorax nos
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, head/headache
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, other
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, neck
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Pain, stomach
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Respiratory, thoracic and mediastinal disorders
Dyspnea
7.7%
4/52 • AE data were collected from Apr 2004 to Apr 2008
Respiratory, thoracic and mediastinal disorders
Cough
3.8%
2/52 • AE data were collected from Apr 2004 to Apr 2008
Respiratory, thoracic and mediastinal disorders
Pleural effusion
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008
Respiratory, thoracic and mediastinal disorders
Fistula, pulmonary, bronchus
1.9%
1/52 • AE data were collected from Apr 2004 to Apr 2008

Other adverse events

Other adverse events
Measure
Sorafenib
n=52 participants at risk
Sorafenib (Nexavar, BAY43-9006) 400 mg twice daily (bid, bis in die) X 28 day cycles
Cardiac disorders
Hypertension
25.0%
13/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Fatigue
61.5%
32/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Weight loss
30.8%
16/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Fever
23.1%
12/52 • AE data were collected from Apr 2004 to Apr 2008
Skin and subcutaneous tissue disorders
Hand-foot skin reaction
36.5%
19/52 • AE data were collected from Apr 2004 to Apr 2008
Skin and subcutaneous tissue disorders
Dermatology - other
26.9%
14/52 • AE data were collected from Apr 2004 to Apr 2008
Skin and subcutaneous tissue disorders
Dry skin
25.0%
13/52 • AE data were collected from Apr 2004 to Apr 2008
Skin and subcutaneous tissue disorders
Pruritus
23.1%
12/52 • AE data were collected from Apr 2004 to Apr 2008
Skin and subcutaneous tissue disorders
Rash/desquamation
21.2%
11/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Diarrhea
51.9%
27/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Anorexia
34.6%
18/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Nausea
36.5%
19/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Vomiting
21.2%
11/52 • AE data were collected from Apr 2004 to Apr 2008
Infections and infestations
Infection - other
23.1%
12/52 • AE data were collected from Apr 2004 to Apr 2008
Blood and lymphatic system disorders
Dermal change
26.9%
14/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, other
36.5%
19/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, chest/thorax nos
26.9%
14/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, head/headache
26.9%
14/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, back
21.2%
11/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, abdomen nos
21.2%
11/52 • AE data were collected from Apr 2004 to Apr 2008
Respiratory, thoracic and mediastinal disorders
Cough
51.9%
27/52 • AE data were collected from Apr 2004 to Apr 2008
Respiratory, thoracic and mediastinal disorders
Dyspnea
44.2%
23/52 • AE data were collected from Apr 2004 to Apr 2008
Respiratory, thoracic and mediastinal disorders
Pulmonary - other
23.1%
12/52 • AE data were collected from Apr 2004 to Apr 2008
Blood and lymphatic system disorders
Hemoglobin
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Cardiac disorders
Hypotension
9.6%
5/52 • AE data were collected from Apr 2004 to Apr 2008
Cardiac disorders
Cardiac general - other
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Skin and subcutaneous tissue disorders
Alopecia
19.2%
10/52 • AE data were collected from Apr 2004 to Apr 2008
Skin and subcutaneous tissue disorders
Nail changes
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Insomnia
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Constitutional symptoms - other
19.2%
10/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Rigors/chills
9.6%
5/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Sweating
13.5%
7/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Constipation
11.5%
6/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Dysphagia
7.7%
4/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Heartburn
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
Mucositis (clinical exam), oral cavitiy
11.5%
6/52 • AE data were collected from Apr 2004 to Apr 2008
Gastrointestinal disorders
GI - other
15.4%
8/52 • AE data were collected from Apr 2004 to Apr 2008
Vascular disorders
Hemorrhage - other
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Vascular disorders
Hemorrhage pulmonary, bronchopulmonary NOS
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Vascular disorders
Hemorrhage pulmonary, nose
7.7%
4/52 • AE data were collected from Apr 2004 to Apr 2008
Musculoskeletal and connective tissue disorders
Musculoskeletal - other
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Musculoskeletal and connective tissue disorders
Gait/walking
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Metabolism and nutrition disorders
AST
7.7%
4/52 • AE data were collected from Apr 2004 to Apr 2008
Metabolism and nutrition disorders
Creatinine
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Metabolism and nutrition disorders
Hyperglycemia
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Metabolism and nutrition disorders
Lipase
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Metabolism and nutrition disorders
Hyponatremia
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Metabolism and nutrition disorders
Metabolic/lab - other
11.5%
6/52 • AE data were collected from Apr 2004 to Apr 2008
Nervous system disorders
Mood alteration, anxiety
7.7%
4/52 • AE data were collected from Apr 2004 to Apr 2008
Nervous system disorders
Dizziness
15.4%
8/52 • AE data were collected from Apr 2004 to Apr 2008
Nervous system disorders
Mood alteration, depression
11.5%
6/52 • AE data were collected from Apr 2004 to Apr 2008
Nervous system disorders
Neurology - other
15.4%
8/52 • AE data were collected from Apr 2004 to Apr 2008
Nervous system disorders
Neuropathy - sensory
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Eye disorders
Ocular - other
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, chest wall
9.6%
5/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, extremity - limb
19.2%
10/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, tumor pain
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, joint
11.5%
6/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, muscle
9.6%
5/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, bone
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, neck
15.4%
8/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, stomach
17.3%
9/52 • AE data were collected from Apr 2004 to Apr 2008
General disorders
Pain, throat/pharynx/larynx
7.7%
4/52 • AE data were collected from Apr 2004 to Apr 2008
Respiratory, thoracic and mediastinal disorders
Hypoxia
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Respiratory, thoracic and mediastinal disorders
Voice changes
11.5%
6/52 • AE data were collected from Apr 2004 to Apr 2008
Renal and urinary disorders
Urinary frequency
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008
Renal and urinary disorders
Renal - other
5.8%
3/52 • AE data were collected from Apr 2004 to Apr 2008

Additional Information

Therapeutic Area Head

BAYER

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: LTE60