Trial Outcomes & Findings for Study of Regorafenib for Urothelial Cancer Following Chemotherapy (UAB 1477) (NCT NCT02459119)

Results Overview

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Death is also considered as progression in the analysis.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

Baseline to 6 months following start of treatment

Results posted on

2020-08-06

Participant Flow

Participant milestones

Participant milestones
Measure	Regorafenib Regorafenib will be administered orally to all patients on study. The drug will be taken once a day for 3 of every 4 week cycle (3 weeks on/1 week off). The dose is 120 mg once daily for the first cycle, then 160 mg once daily from the second cycle if no significant Regorafenib-associated toxicities occur during the first cycle. Drug dosage may be modified if toxicities occur. Patients will undergo up to 4 cycles of treatment and may continue on additional at the discretion of the investigator. Regorafenib: Regorafenib will be packaged as 40 mg tablets in a bottle. Patients will be instructed to maintain a daily medication calendar.
Overall Study STARTED	17
Overall Study COMPLETED	17
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of Regorafenib for Urothelial Cancer Following Chemotherapy (UAB 1477)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Regorafenib n=17 Participants Regorafenib will be administered orally to all patients on study. The drug will be taken once a day for 3 of every 4 week cycle (3 weeks on/1 week off). The dose is 120 mg once daily for the first cycle, then 160 mg once daily from the second cycle if no significant Regorafenib-associated toxicities occur during the first cycle. Drug dosage may be modified if toxicities occur. Patients will undergo up to 4 cycles of treatment and may continue on additional at the discretion of the investigator. Regorafenib: Regorafenib will be packaged as 40 mg tablets in a bottle. Patients will be instructed to maintain a daily medication calendar.
Age, Continuous	67.5 years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	14 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	16 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	17 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to 6 months following start of treatment

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Death is also considered as progression in the analysis.

Outcome measures

Outcome measures
Measure	Regorafenib n=17 Participants Regorafenib will be administered orally to all patients on study. The drug will be taken once a day for 3 of every 4 week cycle (3 weeks on/1 week off). The dose is 120 mg once daily for the first cycle, then 160 mg once daily from the second cycle if no significant Regorafenib-associated toxicities occur during the first cycle. Drug dosage may be modified if toxicities occur. Patients will undergo up to 4 cycles of treatment and may continue on additional at the discretion of the investigator. Regorafenib: Regorafenib will be packaged as 40 mg tablets in a bottle. Patients will be instructed to maintain a daily medication calendar.
Number of Participants With Progression-free Survival at 6 Months	3 Participants

SECONDARY outcome

Timeframe: Every 8 weeks until the time of disease progression upto 2 years

The number of participants showing response at first restaging scan after the start of study treatment. The response will be assessed using tumor measurements which will be documented through CT scans, magnetic resonance imaging (MRI), and x-rays using the Response Evaluation Criteria in Solid Tumors (RECIST). Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR."

Outcome measures

Outcome measures
Measure	Regorafenib n=17 Participants Regorafenib will be administered orally to all patients on study. The drug will be taken once a day for 3 of every 4 week cycle (3 weeks on/1 week off). The dose is 120 mg once daily for the first cycle, then 160 mg once daily from the second cycle if no significant Regorafenib-associated toxicities occur during the first cycle. Drug dosage may be modified if toxicities occur. Patients will undergo up to 4 cycles of treatment and may continue on additional at the discretion of the investigator. Regorafenib: Regorafenib will be packaged as 40 mg tablets in a bottle. Patients will be instructed to maintain a daily medication calendar.
Disease Response Rate	9 Participants

SECONDARY outcome

Timeframe: Baseline to 3 years

Length of subject survival after starting study treatment

Outcome measures

Outcome measures
Measure	Regorafenib n=17 Participants Regorafenib will be administered orally to all patients on study. The drug will be taken once a day for 3 of every 4 week cycle (3 weeks on/1 week off). The dose is 120 mg once daily for the first cycle, then 160 mg once daily from the second cycle if no significant Regorafenib-associated toxicities occur during the first cycle. Drug dosage may be modified if toxicities occur. Patients will undergo up to 4 cycles of treatment and may continue on additional at the discretion of the investigator. Regorafenib: Regorafenib will be packaged as 40 mg tablets in a bottle. Patients will be instructed to maintain a daily medication calendar.
Overall Survival	100 days Standard Error 10.33

SECONDARY outcome

Timeframe: From start of treatment to time of progression or death, assessed up to 6 months

Duration of time from the start of treatment to time of progression or death, whichever comes first. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

Outcome measures

Outcome measures
Measure	Regorafenib n=17 Participants Regorafenib will be administered orally to all patients on study. The drug will be taken once a day for 3 of every 4 week cycle (3 weeks on/1 week off). The dose is 120 mg once daily for the first cycle, then 160 mg once daily from the second cycle if no significant Regorafenib-associated toxicities occur during the first cycle. Drug dosage may be modified if toxicities occur. Patients will undergo up to 4 cycles of treatment and may continue on additional at the discretion of the investigator. Regorafenib: Regorafenib will be packaged as 40 mg tablets in a bottle. Patients will be instructed to maintain a daily medication calendar.
Rate of Progression-free Survival	47.6 days Standard Error 3.6

SECONDARY outcome

Timeframe: At the end of first treatment until 6 months following last treatment, an expected average of 10 months

The Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.0 will be used for assessment of toxicities.

Outcome measures

Outcome measures
Measure	Regorafenib n=17 Participants Regorafenib will be administered orally to all patients on study. The drug will be taken once a day for 3 of every 4 week cycle (3 weeks on/1 week off). The dose is 120 mg once daily for the first cycle, then 160 mg once daily from the second cycle if no significant Regorafenib-associated toxicities occur during the first cycle. Drug dosage may be modified if toxicities occur. Patients will undergo up to 4 cycles of treatment and may continue on additional at the discretion of the investigator. Regorafenib: Regorafenib will be packaged as 40 mg tablets in a bottle. Patients will be instructed to maintain a daily medication calendar.
Number of Participants With Adverse Events	13 Participants

Adverse Events

Regorafenib

Serious events: 7 serious events

Other events: 13 other events

Deaths: 8 deaths

Serious adverse events

Serious adverse events
Measure	Regorafenib n=17 participants at risk Regorafenib will be administered orally to all patients on study. The drug will be taken once a day for 3 of every 4 week cycle (3 weeks on/1 week off). The dose is 120 mg once daily for the first cycle, then 160 mg once daily from the second cycle if no significant Regorafenib-associated toxicities occur during the first cycle. Drug dosage may be modified if toxicities occur. Patients will undergo up to 4 cycles of treatment and may continue on additional at the discretion of the investigator. Regorafenib: Regorafenib will be packaged as 40 mg tablets in a bottle. Patients will be instructed to maintain a daily medication calendar.
Vascular disorders Anemia	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.
Gastrointestinal disorders Diarrhea	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.
Vascular disorders Thrombocytopenia	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.
Musculoskeletal and connective tissue disorders Low Phospohorus	11.8% 2/17 • Number of events 2 • From the date of signing consent form until 6 months after stopping study treatment.
General disorders Fatigue	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.
Renal and urinary disorders Urinary Tract Infection	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.

Other adverse events

Other adverse events
Measure	Regorafenib n=17 participants at risk Regorafenib will be administered orally to all patients on study. The drug will be taken once a day for 3 of every 4 week cycle (3 weeks on/1 week off). The dose is 120 mg once daily for the first cycle, then 160 mg once daily from the second cycle if no significant Regorafenib-associated toxicities occur during the first cycle. Drug dosage may be modified if toxicities occur. Patients will undergo up to 4 cycles of treatment and may continue on additional at the discretion of the investigator. Regorafenib: Regorafenib will be packaged as 40 mg tablets in a bottle. Patients will be instructed to maintain a daily medication calendar.
Musculoskeletal and connective tissue disorders Hand foot syndrome	17.6% 3/17 • Number of events 11 • From the date of signing consent form until 6 months after stopping study treatment.
Vascular disorders Hyponatremia	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.
Vascular disorders Hematuria	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.
Eye disorders Dry eyes	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.
Gastrointestinal disorders Diarrhea	11.8% 2/17 • Number of events 2 • From the date of signing consent form until 6 months after stopping study treatment.
Vascular disorders Epistaxis	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.
Ear and labyrinth disorders Voice hoarseness	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.
Gastrointestinal disorders Vomitting	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.
Cardiac disorders intermittent tachycardia	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.
Musculoskeletal and connective tissue disorders Hypomagnesemia	5.9% 1/17 • Number of events 1 • From the date of signing consent form until 6 months after stopping study treatment.

Additional Information

Dr. Guru Sonpavde, MD

Dana Farber Cancer Institute

Phone: 617-632-2429

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place