Trial Outcomes & Findings for Phase Ib Study of SUnitinib Alternating With REgorafenib in Patients With Metastatic and/or Unresectable GIST (NCT NCT02164240)
NCT ID: NCT02164240
Last Updated: 2021-06-14
Results Overview
Dose-escalation cohort is to determine the frequency and characteristics of DLTs of alternation of sunitinib and regorafenib at each dose level during the first cycle of therapy. Toxicity will be graded accordingly with NCI CTCAE version 4.0
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
14 participants
Primary outcome timeframe
Up to Day 28
Results posted on
2021-06-14
Participant Flow
The "Protocol Enrollment" number reflects the 14 participants who completed the informed consent process, were determined eligible following screening, and were registered in the protocol registration system per study protocol. The "Started in Participant Flow" number reflects the 13 participants who started on the course of study drug treatment following registration (1 of the 14 enrolled participants did not).
Participant milestones
| Measure |
Sunitinib 37.5 and Regorafenib 120
Patients were enrolled and treated with sunitinib 37.5mg daily alternating with regorafenib 120mg daily
|
Sunitinib 37.5 and Regorafenib 160
Patients were enrolled and treated with sunitinib 37.5mg daily alternating with regorafenib 160mg daily
|
Expansion Sunitinib 37.5 Regorafenib 120
Patients were enrolled and treated with sunitinib 37.5mg daily alternating with regorafenib 120mg daily
|
|---|---|---|---|
|
Overall Study
STARTED
|
4
|
5
|
4
|
|
Overall Study
COMPLETED
|
4
|
5
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase Ib Study of SUnitinib Alternating With REgorafenib in Patients With Metastatic and/or Unresectable GIST
Baseline characteristics by cohort
| Measure |
Sunitinib Alternated With Regorafenib
n=13 Participants
Sunitinib: Intervention Description: 3 days of once daily sunitinib alternating with 4 days of once daily regorafenib throughout each 28 day cycle.
|
|---|---|
|
Age, Continuous
|
63.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
12 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
|
PRIMARY outcome
Timeframe: Up to Day 28Dose-escalation cohort is to determine the frequency and characteristics of DLTs of alternation of sunitinib and regorafenib at each dose level during the first cycle of therapy. Toxicity will be graded accordingly with NCI CTCAE version 4.0
Outcome measures
| Measure |
Sunitinib 37.5 and Regorafenib 120
n=4 Participants
Patients were enrolled and treated with sunitinib 37.5 mg daily alternating with regorafenib 120mg daily
|
Sunitinib 37.5 and Regorafenib 160
n=5 Participants
patients were enrolled and treated with sunitinib 37.5 mg daily and regorafenib 160mg daily
|
Expansion Sunitinib 37.5 Regorafenib 120
n=4 Participants
RPD2 determined to be sunitinib 37.5mg daily and regorafenib 120mg daily and therefore, per protocol, the cohort was expanded at this dose level
|
|---|---|---|---|
|
Number of Participants With Serious and Non-Serious Adverse Events
|
4 Participants
|
5 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: 16 weeksPopulation: clinical benefit was analyzed across the entire population based on the pre-planned study design
Percentage of participants who experienced complete response, partial response or stable disease per RECIST 1.1 at 16 weeks
Outcome measures
| Measure |
Sunitinib 37.5 and Regorafenib 120
n=13 Participants
Patients were enrolled and treated with sunitinib 37.5 mg daily alternating with regorafenib 120mg daily
|
Sunitinib 37.5 and Regorafenib 160
patients were enrolled and treated with sunitinib 37.5 mg daily and regorafenib 160mg daily
|
Expansion Sunitinib 37.5 Regorafenib 120
RPD2 determined to be sunitinib 37.5mg daily and regorafenib 120mg daily and therefore, per protocol, the cohort was expanded at this dose level
|
|---|---|---|---|
|
Percentage of Participants With Clinical Benefit
|
0 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: From date of registration until date of protocol-defined progression while on this protocol, assessed up to 6 monthsPopulation: all patients enrolled were analyzed as a single cohort for this endpoint based on pre-planned study design
Non-parametric Kaplan-Meier analysis to assess median progression free survival (mPFS)
Outcome measures
| Measure |
Sunitinib 37.5 and Regorafenib 120
n=13 Participants
Patients were enrolled and treated with sunitinib 37.5 mg daily alternating with regorafenib 120mg daily
|
Sunitinib 37.5 and Regorafenib 160
patients were enrolled and treated with sunitinib 37.5 mg daily and regorafenib 160mg daily
|
Expansion Sunitinib 37.5 Regorafenib 120
RPD2 determined to be sunitinib 37.5mg daily and regorafenib 120mg daily and therefore, per protocol, the cohort was expanded at this dose level
|
|---|---|---|---|
|
Median Progression Free Survival (mPFS)
|
1.9 months
Interval 1.4 to 3.6
|
—
|
—
|
Adverse Events
Cohort 1: SuRe120
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Cohort 2: SuRe160
Serious events: 3 serious events
Other events: 5 other events
Deaths: 0 deaths
Cohort 3: ExpSuRe120
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1: SuRe120
n=4 participants at risk
Patients were enrolled and treated with sunitinib 37.5mg/d alternating with regorafenib 120 mg/d
|
Cohort 2: SuRe160
n=5 participants at risk
Patients were enrolled and treated with sunitinib 37.5mg/d alternating with regorafenib 160 mg/d
|
Cohort 3: ExpSuRe120
n=4 participants at risk
Patients were enrolled and treated with sunitinib 37.5mg/d alternating with regorafenib 120 mg/d
|
|---|---|---|---|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
0.00%
0/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
0.00%
0/4
|
40.0%
2/5
|
0.00%
0/4
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
25.0%
1/4
|
0.00%
0/5
|
25.0%
1/4
|
|
Vascular disorders
Hypertension
|
25.0%
1/4
|
20.0%
1/5
|
25.0%
1/4
|
Other adverse events
| Measure |
Cohort 1: SuRe120
n=4 participants at risk
Patients were enrolled and treated with sunitinib 37.5mg/d alternating with regorafenib 120 mg/d
|
Cohort 2: SuRe160
n=5 participants at risk
Patients were enrolled and treated with sunitinib 37.5mg/d alternating with regorafenib 160 mg/d
|
Cohort 3: ExpSuRe120
n=4 participants at risk
Patients were enrolled and treated with sunitinib 37.5mg/d alternating with regorafenib 120 mg/d
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
50.0%
2/4
|
0.00%
0/5
|
0.00%
0/4
|
|
Gastrointestinal disorders
Bloating
|
25.0%
1/4
|
0.00%
0/5
|
0.00%
0/4
|
|
Gastrointestinal disorders
Constipation
|
25.0%
1/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Gastrointestinal disorders
Diarrhea
|
100.0%
4/4
|
60.0%
3/5
|
0.00%
0/4
|
|
Gastrointestinal disorders
Dry mouth
|
0.00%
0/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Gastrointestinal disorders
Flatulence
|
0.00%
0/4
|
0.00%
0/5
|
50.0%
2/4
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
0.00%
0/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
0.00%
0/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Gastrointestinal disorders
Mucositis oral
|
0.00%
0/4
|
40.0%
2/5
|
25.0%
1/4
|
|
Gastrointestinal disorders
Nausea
|
25.0%
1/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Gastrointestinal disorders
Oral dysesthesia
|
0.00%
0/4
|
20.0%
1/5
|
0.00%
0/4
|
|
General disorders
Chills
|
0.00%
0/4
|
20.0%
1/5
|
25.0%
1/4
|
|
General disorders
Fatigue
|
100.0%
4/4
|
80.0%
4/5
|
100.0%
4/4
|
|
General disorders
Pain
|
0.00%
0/4
|
0.00%
0/5
|
25.0%
1/4
|
|
Hepatobiliary disorders
Hepatic hemorrhage
|
0.00%
0/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Infections and infestations
Mucosal infection
|
0.00%
0/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Investigations
Investigations - Other, specify
|
0.00%
0/4
|
0.00%
0/5
|
25.0%
1/4
|
|
Investigations
Weight loss
|
50.0%
2/4
|
80.0%
4/5
|
50.0%
2/4
|
|
Metabolism and nutrition disorders
Anorexia
|
25.0%
1/4
|
80.0%
4/5
|
25.0%
1/4
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/4
|
0.00%
0/5
|
25.0%
1/4
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other, specify
|
0.00%
0/4
|
40.0%
2/5
|
0.00%
0/4
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
25.0%
1/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Nervous system disorders
Dysgeusia
|
0.00%
0/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Nervous system disorders
Headache
|
0.00%
0/4
|
0.00%
0/5
|
25.0%
1/4
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
50.0%
2/4
|
40.0%
2/5
|
25.0%
1/4
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
50.0%
2/4
|
60.0%
3/5
|
0.00%
0/4
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
25.0%
1/4
|
0.00%
0/5
|
0.00%
0/4
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
0.00%
0/4
|
40.0%
2/5
|
25.0%
1/4
|
|
Vascular disorders
Flushing
|
0.00%
0/4
|
0.00%
0/5
|
25.0%
1/4
|
|
Vascular disorders
Hot flashes
|
0.00%
0/4
|
20.0%
1/5
|
0.00%
0/4
|
|
Vascular disorders
Hypertension
|
0.00%
0/4
|
40.0%
2/5
|
25.0%
1/4
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place