Trial Outcomes & Findings for Sorafenib in Treating Patients With Regional or Metastatic Cancer of the Urothelium (NCT NCT00112905)

Last Updated: 2014-05-30

Results Overview

Survival estimate from the Kaplan-Meier curve of the proportion of patients alive and progression-free at 4 months. Progression-free survival is defined as the time from registration to progression or death, whichever occurs first. Progression is defined as at least a 20% increase in the sum of the longest diameters of target lesions, taking as reference the smallest sum longest diameter recorded since the baseline measurements, or the appearance of one or more new lesion(s) or unequivocal progression of existing nontarget lesions.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

27 participants

Primary outcome timeframe

Assessed every cycle while on treatment; after being off-treatment, assessed every 3 months for 2 years, then every 6 months for 1 year.

Results posted on

2014-05-30

Participant Flow

The study was activated on 10/26/2005. The TCC (transitional cell carcinoma) cohort was suspended for an efficacy evaluation on 10/30/2006 after reaching its first stage accrual goal. A total of 27 patients entered the TCC cohort, and no patient entered the non-TCC cohort. The study was terminated on March 10th, 2008.

Participant milestones

Participant milestones
Measure	TCC (Transitional Cell Carcinoma) Cohort Sorafenib was administered at a dose of 400 mg orally twice daily (total daily dose 800 mg) for eight weeks as one cycle. Patients swallowed the tablets whole with approximately 250 ml (8 oz.) of water each morning and evening (i.e., 12-hourly), with or without food. If sorafenib was taken with meals, patients were instructed to take sorafenib tosylate with a moderate to low-fat meal, as a high fat meal caused a decrease in absorption in previous studies. Patients were instructed to keep a pill diary and record the pills they took each day.
Overall Study STARTED	27
Overall Study Eligible and Treated	22
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	12

Reasons for withdrawal

Reasons for withdrawal
Measure	TCC (Transitional Cell Carcinoma) Cohort Sorafenib was administered at a dose of 400 mg orally twice daily (total daily dose 800 mg) for eight weeks as one cycle. Patients swallowed the tablets whole with approximately 250 ml (8 oz.) of water each morning and evening (i.e., 12-hourly), with or without food. If sorafenib was taken with meals, patients were instructed to take sorafenib tosylate with a moderate to low-fat meal, as a high fat meal caused a decrease in absorption in previous studies. Patients were instructed to keep a pill diary and record the pills they took each day.
Overall Study Adverse Event	4
Overall Study Death	1
Overall Study Ineligibility	5
Overall Study Alternative treatment	1
Overall Study performance status was deteiorating	1

Baseline Characteristics

Sorafenib in Treating Patients With Regional or Metastatic Cancer of the Urothelium

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	TCC Cohort n=22 Participants Only eligible and treated patients are included in the analysis. BAY 43-9006 was administered at a dose of 400 mg orally twice daily (total daily dose 800 mg) for eight weeks as one cycle. Patients swallowed the tablets whole with approximately 250 ml (8 oz.) of water each morning and evening (i.e., 12-hourly), with or without food. If Bay 43-9006 was taken with meals, patients were instructed to take BAY 43-9006 tosylate with a moderate to low-fat meal, as a high fat meal caused a decrease in absorption in previous studies.
Age, Continuous	66 years n=93 Participants
Sex: Female, Male Female	8 Participants n=93 Participants
Sex: Female, Male Male	14 Participants n=93 Participants

PRIMARY outcome

Timeframe: Assessed every cycle while on treatment; after being off-treatment, assessed every 3 months for 2 years, then every 6 months for 1 year.

Population: Per protocol, the primary analysis included eligible patients who started protocol treatment.

Outcome measures

Outcome measures
Measure	TCC Cohort n=22 Participants Only eligible and treated patients are included in the analysis. BAY 43-9006 was administered at a dose of 400 mg orally twice daily (total daily dose 800 mg) for eight weeks as one cycle. Patients swallowed the tablets whole with approximately 250 ml (8 oz.) of water each morning and evening (i.e., 12-hourly), with or without food. If Bay 43-9006 was taken with meals, patients were instructed to take BAY 43-9006 tosylate with a moderate to low-fat meal, as a high fat meal caused a decrease in absorption in previous studies.
Kaplan-Meier Estimate of Progression-free Survival at 4 Months	11 Percentage of Participants Interval 0.0 to 23.0

SECONDARY outcome

Timeframe: Assessed every cycle while on treatment; after being off-treatment, assessed every 3 months for 2 years, then every 6 months for 1 year.

Population: Only eligible and treated patients are included in this analysis.

Time from registration to the earlier of disease progression or death. Patients alive and progression-free at last follow-up were censored. Progression is defined as at least a 20% increase in the sum of the longest diameters of target lesions, taking as reference the smallest sum longest diameter recorded since the baseline measurements, or the appearance of one or more new lesion(s) or unequivocal progression of existing nontarget lesions.

Outcome measures

Outcome measures
Measure	TCC Cohort n=22 Participants Only eligible and treated patients are included in the analysis. BAY 43-9006 was administered at a dose of 400 mg orally twice daily (total daily dose 800 mg) for eight weeks as one cycle. Patients swallowed the tablets whole with approximately 250 ml (8 oz.) of water each morning and evening (i.e., 12-hourly), with or without food. If Bay 43-9006 was taken with meals, patients were instructed to take BAY 43-9006 tosylate with a moderate to low-fat meal, as a high fat meal caused a decrease in absorption in previous studies.
Progression-free Survival	2.2 Months Interval 1.8 to 3.7

SECONDARY outcome

Timeframe: Assessed every cycle while on treatment; after being off-treatment, assessed every 3 months for 2 years, then every 6 months for 1 year.

Population: Only eligible and treated patients are included in this analysis.

Time from registration to death. Patients alive at last follow-up were censored.

Outcome measures

Outcome measures
Measure	TCC Cohort n=22 Participants Only eligible and treated patients are included in the analysis. BAY 43-9006 was administered at a dose of 400 mg orally twice daily (total daily dose 800 mg) for eight weeks as one cycle. Patients swallowed the tablets whole with approximately 250 ml (8 oz.) of water each morning and evening (i.e., 12-hourly), with or without food. If Bay 43-9006 was taken with meals, patients were instructed to take BAY 43-9006 tosylate with a moderate to low-fat meal, as a high fat meal caused a decrease in absorption in previous studies.
Overall Survival	6.8 Months Interval 5.7 to 8.5

SECONDARY outcome

Timeframe: Assessed every cycle while on treatment; after being off-treatment, assessed every 3 months for 2 years, then every 6 months for 1 year.

Population: Only eligible and treated patients are included in the analysis.

This outcome measure reports the best response a patient has ever experienced. \<Target Lesions\> Complete Response (CR): The disappearance of all target lesions, confirmed by assessments \>=4 weeks (wks) later. Partial Response: \>=30% decrease in the sum of the longest diameters of target lesions from baseline, confirmed by assessments \>=4 wks later. Progressive Disease (PD): \>=20% increase in the sum of the longest diameters of target lesions from the smallest sum longest diameter since baseline, or the appearance of new lesions. Stable Disease (SD): Neither response criteria nor progressive disease criteria are met for \>=8 wks. \<Nontarget Lesions\> CR: The disappearance of all nontarget lesions and normalization of tumor marker levels, confirmed by assessments \>=4 wks later. SD: Persistence of nontarget lesions or maintenance of tumor marker levels above the normal limits for \>=8 wks. PD: The appearance of new lesions or unequivocal progression of existing lesions.

Outcome measures

Outcome measures
Measure	TCC Cohort n=22 Participants Only eligible and treated patients are included in the analysis. BAY 43-9006 was administered at a dose of 400 mg orally twice daily (total daily dose 800 mg) for eight weeks as one cycle. Patients swallowed the tablets whole with approximately 250 ml (8 oz.) of water each morning and evening (i.e., 12-hourly), with or without food. If Bay 43-9006 was taken with meals, patients were instructed to take BAY 43-9006 tosylate with a moderate to low-fat meal, as a high fat meal caused a decrease in absorption in previous studies.
Best Overall Response by RECIST Complete response	0 Participants
Best Overall Response by RECIST Partial response	0 Participants
Best Overall Response by RECIST Stable disease	3 Participants
Best Overall Response by RECIST Progression	9 Participants
Best Overall Response by RECIST Unevaluable	10 Participants

Adverse Events

TCC Cohort

Serious events: 15 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	TCC Cohort n=27 participants at risk Only eligible and treated patients are included in the analysis. BAY 43-9006 was administered at a dose of 400 mg orally twice daily (total daily dose 800 mg) for eight weeks as one cycle. Patients swallowed the tablets whole with approximately 250 ml (8 oz.) of water each morning and evening (i.e., 12-hourly), with or without food. If Bay 43-9006 was taken with meals, patients were instructed to take BAY 43-9006 tosylate with a moderate to low-fat meal, as a high fat meal caused a decrease in absorption in previous studies.
Vascular disorders Hypertension	3.7% 1/27 • Assessed every 8 weeks
General disorders Fatique	18.5% 5/27 • Assessed every 8 weeks
Skin and subcutaneous tissue disorders Rash/desquamation	7.4% 2/27 • Assessed every 8 weeks
Skin and subcutaneous tissue disorders Hand-foot reaction	18.5% 5/27 • Assessed every 8 weeks
Endocrine disorders Hyperthyroidism, thyrotoxicosis	3.7% 1/27 • Assessed every 8 weeks
Metabolism and nutrition disorders Anorexia	3.7% 1/27 • Assessed every 8 weeks
Metabolism and nutrition disorders Dehydration	3.7% 1/27 • Assessed every 8 weeks
Gastrointestinal disorders Muco/stomatitis (symptom) oral cavity	3.7% 1/27 • Assessed every 8 weeks
Musculoskeletal and connective tissue disorders Nonneuropathic generalized weakness	3.7% 1/27 • Assessed every 8 weeks
Nervous system disorders Dizziness	3.7% 1/27 • Assessed every 8 weeks
Vascular disorders Thrombosis/thrombus/embolism	7.4% 2/27 • Assessed every 8 weeks

Other adverse events

Other adverse events
Measure	TCC Cohort n=27 participants at risk Only eligible and treated patients are included in the analysis. BAY 43-9006 was administered at a dose of 400 mg orally twice daily (total daily dose 800 mg) for eight weeks as one cycle. Patients swallowed the tablets whole with approximately 250 ml (8 oz.) of water each morning and evening (i.e., 12-hourly), with or without food. If Bay 43-9006 was taken with meals, patients were instructed to take BAY 43-9006 tosylate with a moderate to low-fat meal, as a high fat meal caused a decrease in absorption in previous studies.
Vascular disorders Hypertension	29.6% 8/27 • Assessed every 8 weeks
General disorders Fatigue	51.9% 14/27 • Assessed every 8 weeks
Investigations Weight loss	18.5% 5/27 • Assessed every 8 weeks
Skin and subcutaneous tissue disorders Dry skin	7.4% 2/27 • Assessed every 8 weeks
Skin and subcutaneous tissue disorders Rash/desquamation	18.5% 5/27 • Assessed every 8 weeks
Skin and subcutaneous tissue disorders Hand-foot reaction	22.2% 6/27 • Assessed every 8 weeks
Metabolism and nutrition disorders Anorexia	37.0% 10/27 • Assessed every 8 weeks
Gastrointestinal disorders Constipation	18.5% 5/27 • Assessed every 8 weeks
Gastrointestinal disorders Diarrhea w/o prior colostomy	25.9% 7/27 • Assessed every 8 weeks
Gastrointestinal disorders Mucositis/stomatitis (symptom) oral cavity	11.1% 3/27 • Assessed every 8 weeks
Gastrointestinal disorders Nausea	33.3% 9/27 • Assessed every 8 weeks
Nervous system disorders Taste disturbance	7.4% 2/27 • Assessed every 8 weeks
Gastrointestinal disorders Vomiting	18.5% 5/27 • Assessed every 8 weeks
Investigations ALT increased	7.4% 2/27 • Assessed every 8 weeks
Musculoskeletal and connective tissue disorders Back pain	7.4% 2/27 • Assessed every 8 weeks

Additional Information

Study Statistician

ECOG Statistical Office

Phone: 617-632-3012

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60