Trial Outcomes & Findings for Regorafenib in Treating Patients With Advanced or Metastatic Neuroendocrine Tumors (NCT NCT02259725)

Last Updated: 2021-12-15

Results Overview

Two parallel Simon's 2-stage phase II trials will be conducted to evaluate the efficacy of regorafenib in patients with advanced carcinoid (cohort A) or pancreatic islet cell tumors (cohort B). Will be summarized as a proportion of patients who are alive and progression-free among all patients in the primary data analysis set. The 95% confidence intervals (CIs) will be calculated using the Wilson method. Will be analyzed using Kaplan-Meier (KM) curves. The median PFS and 95% CIs will be calculated. The probability of 6-month PFS will be estimated from the KM curve too.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Time from start of treatment to time of progression or death on study whichever comes first, assessed at 6 months

Results posted on

2021-12-15

Participant Flow

The study began recruiting in August 2016 and ended in January 2019. All subjects were seen and treated either at USC Norris Comprehensive Cancer Center or at LAC+USC Medical Center.

There were no pre-assignment criteria. All subjects were given the same treatment.

Participant milestones

Participant milestones
Measure	Treatment (Regorafenib) Patients receive regorafenib PO QD on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. regorafenib: Given PO laboratory biomarker analysis: Correlative studies
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Regorafenib in Treating Patients With Advanced or Metastatic Neuroendocrine Tumors

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Regorafenib) n=3 Participants Patients receive regorafenib PO QD on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. regorafenib: Given PO laboratory biomarker analysis: Correlative studies
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 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	0 Participants n=5 Participants
Race (NIH/OMB) White	3 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	3 participants n=5 Participants

PRIMARY outcome

Timeframe: Time from start of treatment to time of progression or death on study whichever comes first, assessed at 6 months

Population: No analysis done. Only 3 subjects were enrolled out of accrual goal of 24 subjects.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 4 years

Population: No analysis was done. Only 3 subjects were enrolled out of accrual goal of 24 subjects.

Will be calculated as a proportion of patients who have either a complete or partial response among all patients in the primary data analysis set. The 95% CIs will be given.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From start of treatment until death due to any cause, assessed up to 4 years

Population: Due to insufficient accrual (only 3 accrued), no analysis was performed.

The 95% CIs will be calculated using the Wilson method. Will be analyzed using KM curves.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 4 years

Population: Data not collected

Toxicity profile will be summarized by attribution: regorafenib-related and all reported, course: course 1 and all courses, type, and grade: grade 1-2, 3-4, and 5.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 4 years

Population: Analysis not done. Only 3 subjects were enrolled out of the accrual goal of subjects.

The associations between biomarkers and clinical outcomes (6-month PFS, response, PFS, and overall survival) will be analyzed in univariate analysis first using appropriate methods. Multivariable analyses will be conducted to evaluate the independent effect of a marker on clinical outcome. All tests will be two-sided at a significance level of 0.05. P values will be adjusted for multiple comparisons.

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Regorafenib)

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Regorafenib) n=3 participants at risk Patients receive regorafenib PO QD on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. regorafenib: Given PO laboratory biomarker analysis: Correlative studies
Gastrointestinal disorders Constipation	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Gastrointestinal disorders Diarrhea	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Investigations Alanine aminotransferase increased	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Investigations Aspartate aminotransferase increased	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Investigations Blood bilirubin increased	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.

Other adverse events

Other adverse events
Measure	Treatment (Regorafenib) n=3 participants at risk Patients receive regorafenib PO QD on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. regorafenib: Given PO laboratory biomarker analysis: Correlative studies
Blood and lymphatic system disorders Anemia	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Gastrointestinal disorders Abdominal pain	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Gastrointestinal disorders Bloating	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Gastrointestinal disorders Diarrhea	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Gastrointestinal disorders Mucositis oral	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Gastrointestinal disorders Nausea	100.0% 3/3 • Number of events 3 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Gastrointestinal disorders Stomach pain	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Gastrointestinal disorders Vomiting	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
General disorders Edema limbs	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
General disorders Fatigue	33.3% 1/3 • Number of events 3 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Infections and infestations Paronychia	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Investigations Alkaline phosphatase increased	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Investigations Blood bilirubin increased	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Investigations Lipase increased	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Metabolism and nutrition disorders Dehydration	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Metabolism and nutrition disorders Hypercalcemia	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Metabolism and nutrition disorders Hyperuricemia	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Metabolism and nutrition disorders Hypocalcemia	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Musculoskeletal and connective tissue disorders Flank pain	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Musculoskeletal and connective tissue disorders Myalgia	66.7% 2/3 • Number of events 3 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Musculoskeletal and connective tissue disorders Pain in extremity	33.3% 1/3 • Number of events 3 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Nervous system disorders Dysgeusia	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Reproductive system and breast disorders Testicular pain	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Reproductive system and breast disorders Hoarseness	66.7% 2/3 • Number of events 2 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Reproductive system and breast disorders Voice alteration	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Skin and subcutaneous tissue disorders Alopecia	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Skin and subcutaneous tissue disorders Dry skin	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysesthesia syndrome	66.7% 2/3 • Number of events 2 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.
Vascular disorders Hypertension	66.7% 2/3 • Number of events 2 • Adverse events were collected from the time the subject received the initial study drug administration and continued throughout the study until 30 days after the last dose or until resolution of adverse event if beyond 30 days after last dose. Adverse events are assessed during regularly scheduled follow up.

Additional Information

Victoria Soto

USC / Norris Comprehensive Cancer Center

Phone: 3238653000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place