Trial Outcomes & Findings for Regorafenib in Treating Patients With Locally Advanced Cancer of the Esophagus or Gastroesophageal Junction Who Have Completed Chemoradiation Therapy and Surgery (NCT NCT02234180)

Results Overview

Disease free survival (DFS) is defined as the time from randomization to the first of either disease recurrence or death from any cause. The distribution of DFS will be estimated using the Kaplan Meier method.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Time from randomization to the first of either disease recurrence or death from any cause, assessed up to 1 year and 10 months

Results posted on

2018-10-17

Participant Flow

Participant milestones

Participant milestones
Measure	Arm I (Regorafenib) Within 6-12 weeks after surgery, patients will receive regorafenib PO QD on days 1-21. Courses repeat every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity.	Arm II (Placebo) Within 6-12 weeks after surgery, patients will receive placebo PO QD on days 1-21. Courses repeat every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity.
Overall Study STARTED	2	1
Overall Study COMPLETED	2	1
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Regorafenib in Treating Patients With Locally Advanced Cancer of the Esophagus or Gastroesophageal Junction Who Have Completed Chemoradiation Therapy and Surgery

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm I/II (Regorafenib/Placebo) n=3 Participants Within 6-12 weeks after surgery, patients receive regorafenib OR placebo PO QD on days 1-21. Courses repeat every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity.
Age, Continuous	54.0 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: Time from randomization to the first of either disease recurrence or death from any cause, assessed up to 1 year and 10 months

Population: Only 1 patient was registered to the placebo arm. Due to protected health information, listing only 1 patient's results is contraindicated. The data below is descriptive and shouldn't be trusted because of the small sample size.

Disease free survival (DFS) is defined as the time from randomization to the first of either disease recurrence or death from any cause. The distribution of DFS will be estimated using the Kaplan Meier method.

Outcome measures

Outcome measures
Measure	Arm I/II (Regorafenib/Placebo) n=3 Participants Within 6-12 weeks after surgery, patients receive regorafenib OR placebo PO QD on days 1-21. Courses repeat every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity.
Disease Free Survival (DFS)	4.83 months Interval 2.27 to 9.89

SECONDARY outcome

Timeframe: Up to 1 year and 10 months

Population: Only 1 patient was registered to the placebo arm. Due to protected health information, listing only 1 patient's results is contraindicated. The data below is descriptive and shouldn't be trusted because of the small sample size.

The maximum grade for each type of toxicity will be recorded for each patient, and frequency tables will be reviewed to determine toxicity patterns within patient groups. In addition, we will review all adverse event data that is graded as 3, 4, or 5 and classified as either "unrelated" or "unlikely to be related" to study treatment in the event of an actual relationship developing. The overall toxicity rates (percentages) for grade 3 or higher adverse events considered at least possibly related to treatment are reported below.

Outcome measures

Outcome measures
Measure	Arm I/II (Regorafenib/Placebo) n=3 Participants Within 6-12 weeks after surgery, patients receive regorafenib OR placebo PO QD on days 1-21. Courses repeat every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity.
Toxicity, Assessed Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 (v4)	0 percentage of grade 3+ AEs

SECONDARY outcome

Timeframe: Time from randomization to death due to any cause, assessed up to 1 year and 10 months

Population: Only 1 patient was registered to the placebo arm. Due to protected health information, listing only 1 patient's results is contraindicated. The data below is descriptive and shouldn't be trusted because of the small sample size.

Overall survival (OS) is defined as the time from randomization to death due to any cause.

Outcome measures

Outcome measures
Measure	Arm I/II (Regorafenib/Placebo) n=3 Participants Within 6-12 weeks after surgery, patients receive regorafenib OR placebo PO QD on days 1-21. Courses repeat every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity.
Overall Survival (OS)	NA months No OS events observed among the patients, thus no OS median and 95% confidence interval is available.

Adverse Events

Arm I/II (Regorafenib/Placebo)

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Arm I/II (Regorafenib/Placebo) n=3 participants at risk Within 6-12 weeks after surgery, patients receive regorafenib OR placebo PO QD on days 1-21. Courses repeat every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity.
Vascular disorders Thromboembolic event	33.3% 1/3 • Number of events 1 • Up to 1 year and 10 months The descriptions \& grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for AE reporting. Each CTCAE term in the current version is a unique representation of a specific event used for medical documentation \& scientific analysis \& is a single MedDRA Lowest Level Term (LLT). Only 1 patient was registered to the placebo arm. Due to protected health information, listing only 1 patient's results is contraindicated.

Other adverse events

Other adverse events
Measure	Arm I/II (Regorafenib/Placebo) n=3 participants at risk Within 6-12 weeks after surgery, patients receive regorafenib OR placebo PO QD on days 1-21. Courses repeat every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity.
Gastrointestinal disorders Diarrhea	66.7% 2/3 • Number of events 6 • Up to 1 year and 10 months The descriptions \& grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for AE reporting. Each CTCAE term in the current version is a unique representation of a specific event used for medical documentation \& scientific analysis \& is a single MedDRA Lowest Level Term (LLT). Only 1 patient was registered to the placebo arm. Due to protected health information, listing only 1 patient's results is contraindicated.
Gastrointestinal disorders Vomiting	33.3% 1/3 • Number of events 2 • Up to 1 year and 10 months The descriptions \& grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for AE reporting. Each CTCAE term in the current version is a unique representation of a specific event used for medical documentation \& scientific analysis \& is a single MedDRA Lowest Level Term (LLT). Only 1 patient was registered to the placebo arm. Due to protected health information, listing only 1 patient's results is contraindicated.
General disorders Fatigue	33.3% 1/3 • Number of events 3 • Up to 1 year and 10 months The descriptions \& grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for AE reporting. Each CTCAE term in the current version is a unique representation of a specific event used for medical documentation \& scientific analysis \& is a single MedDRA Lowest Level Term (LLT). Only 1 patient was registered to the placebo arm. Due to protected health information, listing only 1 patient's results is contraindicated.
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysesthesia syndrome	66.7% 2/3 • Number of events 7 • Up to 1 year and 10 months The descriptions \& grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for AE reporting. Each CTCAE term in the current version is a unique representation of a specific event used for medical documentation \& scientific analysis \& is a single MedDRA Lowest Level Term (LLT). Only 1 patient was registered to the placebo arm. Due to protected health information, listing only 1 patient's results is contraindicated.
Vascular disorders Hypertension	66.7% 2/3 • Number of events 6 • Up to 1 year and 10 months The descriptions \& grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for AE reporting. Each CTCAE term in the current version is a unique representation of a specific event used for medical documentation \& scientific analysis \& is a single MedDRA Lowest Level Term (LLT). Only 1 patient was registered to the placebo arm. Due to protected health information, listing only 1 patient's results is contraindicated.

Additional Information

Yelena Y. Janjigian MD

Memorial Sloan-Kettering Cancer Center

Phone: 646-888-4186

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place