Trial Outcomes & Findings for Sorafenib in Treating Patients With Malignant Gastrointestinal Stromal Tumor That Progressed During or After Previous Treatment With Imatinib Mesylate and Sunitinib Malate (NCT NCT00265798)

Results Overview

Objective response (complete response (CR)+ partial response (PR)) will be evaluated using Response Evaluation Criteria in Solid Tumors (RECIST) criteria. CR is the disappearance of all target lesions. PR requires at least a 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD. Computed Tomography (CT) scans for disease reassessment will be obtained pre-therapy and every 8 weeks. In addition to a baseline scan, confirmatory scans will also be obtained 4 weeks following initial documentation of objective response.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

38 participants

Primary outcome timeframe

Up to 5 years

Results posted on

2025-08-20

Participant Flow

Patients were enrolled from 6 centers between 2006 and 2009

Participant milestones

Participant milestones
Measure	Sorafenib Patients receive oral Sorafenib 400mg twice daily on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Overall Study STARTED	38
Overall Study COMPLETED	38
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Sorafenib in Treating Patients With Malignant Gastrointestinal Stromal Tumor That Progressed During or After Previous Treatment With Imatinib Mesylate and Sunitinib Malate

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sorafenib n=38 Participants Patients receive oral Sorafenib 400mg twice daily on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Age, Continuous	57 years n=5 Participants
Sex: Female, Male Female	17 Participants n=5 Participants
Sex: Female, Male Male	21 Participants n=5 Participants
Region of Enrollment United States	38 participants n=5 Participants
Study Cohort Imatinib-resistant	6 participants n=5 Participants
Study Cohort Imatinib- and sunitinib-resistant	32 participants n=5 Participants
Performance Status 0	18 participants n=5 Participants
Performance Status 1	18 participants n=5 Participants
Performance Status 2	2 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 5 years

Objective response (complete response (CR)+ partial response (PR)) will be evaluated using Response Evaluation Criteria in Solid Tumors (RECIST) criteria. CR is the disappearance of all target lesions. PR requires at least a 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD. Computed Tomography (CT) scans for disease reassessment will be obtained pre-therapy and every 8 weeks. In addition to a baseline scan, confirmatory scans will also be obtained 4 weeks following initial documentation of objective response.

Outcome measures

Outcome measures
Measure	Sorafenib n=38 Participants Patients receive oral Sorafenib 400mg twice daily on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Objective Response Rate	13 percentage of partcipants Interval 4.0 to 28.0

SECONDARY outcome

Timeframe: Up to 5 years

Progression-free survival will be defined as time from the start of treatment until progression (documented according to Response Evaluation Criteria in Solid Tumors (RECIST) criteria and defined as at least a 20% increase in the sum of the longest diameter of target lesions) or death, whichever comes first. CT scans for disease reassessment will be obtained pre-therapy and every 8 weeks.

Outcome measures

Outcome measures
Measure	Sorafenib n=38 Participants Patients receive oral Sorafenib 400mg twice daily on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Progression-free Survival	5.2 months Interval 3.4 to 7.4

SECONDARY outcome

Timeframe: Up to 5 years

Overall survival will be defined as time from the start of treatment until death from any cause.

Outcome measures

Outcome measures
Measure	Sorafenib n=38 Participants Patients receive oral Sorafenib 400mg twice daily on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Overall Survival	11.6 months Interval 8.8 to 14.3

Adverse Events

Sorafenib

Serious events: 5 serious events

Other events: 23 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Sorafenib n=38 participants at risk Patients receive oral Sorafenib 400mg twice daily on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Vascular disorders Thrombosis	2.6% 1/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported
Gastrointestinal disorders Gastrointestinal hemorrhage	5.3% 2/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported
Skin and subcutaneous tissue disorders Rash	2.6% 1/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported
Gastrointestinal disorders Diarrhea	2.6% 1/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported
Metabolism and nutrition disorders Dehydration	2.6% 1/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported
Vascular disorders Hypertension	2.6% 1/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported
Metabolism and nutrition disorders Hypophosphatemia	2.6% 1/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported

Other adverse events

Other adverse events
Measure	Sorafenib n=38 participants at risk Patients receive oral Sorafenib 400mg twice daily on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Gastrointestinal disorders Diarrhea	5.3% 2/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported
Metabolism and nutrition disorders Hypophosphatemia	7.9% 3/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported
Investigations Alkaline phosphatase increased	5.3% 2/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported
Vascular disorders Hypertension	18.4% 7/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysesthesia syndrome	44.7% 17/38 • Adverse events were monitored over the course of treatment Adverse events of grade 3 or higher at least possibly related to treatment are reported

Additional Information

Dr. Hedy L. Kindler

University of Chicago

Phone: 773-702-0360

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60