Study of Regorafenib as a 3rd-line or Beyond Treatment for Gastrointestinal Stromal Tumors (GIST)

Draft scope assessment: unclear. Attached because the study's own record describes metastatic disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full. Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.

Read the full study record and all posted results →

Linked outcomes

Measures are not ranked. Open each original source section for treatment groups, denominators, units and results.

primary · POSTED

Progression-free Survival (PFS) was defined as the time from date of randomization to radiological disease progression or death due to any cause, whichever occurs first. PFS was based on central radiological assessment using modified RECIST (Response Evaluation Criteria in Solid Tumors) v.1.1. Progression is defined as at least a 20% increase in the sum of diameters of target lesions taking as reference the smallest sum on study; or unequivocal progression of existing non-target lesions; or appearance of new lesions. Subjects without progression or death at the time of analysis were censored at their last date of tumor evaluation. Results are based on central evaluation.

Full Analysis Set (FAS) - defined as all randomized participants.

→

secondary · POSTED

Overall Survival (OS) was defined as the time from date of randomization to death due to any cause. Subjects still alive at the time of analysis were censored at their date of last contact. Median OS was not observed at the time of PFS analysis and first analysis of OS, therefore only the proportion of death events was reported in the results posting system. This approach was maintained for the subsequent updates in the results posting system.

Full Analysis Set (FAS). 58 (87.9%) patients in placebo group and 91 (68.4%) patients in regorafenib had started open-label treatment with regorafenib before time of final database cutoff 08 Jun 2015

→

secondary · POSTED

Time to progression (TTP) was defined as the time from date of randomization to disease progression (based on central radiological assessment using modified RECIST \[Response Evaluation Criteria in Solid Tumors\] v.1.1). Progression is defined as at least a 20% increase in the sum of diameters of target lesions taking as reference the smallest sum on study; or unequivocal progression of existing non-target lesions; or appearance of new lesions. Subjects without progression at the time of analysis were censored at their last date of tumor evaluation. Results are based on central evaluation.

Full Analysis Set (FAS)

→

secondary · POSTED

Tumor Response of a subject was defined as the best tumor response (Complete Response \[CR: disappearance of all clinical and radiological evidence of tumor (both target and non-target).\], Partial Response \[PR: at least a 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum, no unequivocal progression of existing non-target lesions, and no appearance of new lesions.\], Stable Disease \[SD: steady state of disease. Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, no unequivocal progression of existing non-target lesions, and no appearance of new lesions.\], or Progressive Disease \[PD: at least a 20% increase in the sum of diameters of target lesions taking as reference the smallest sum on study or unequivocal progression of existing non-target lesions, or appearance of new lesions.\]) observed during the trial period and assessed according to RECIST v1.1 criteria. Results are based on central evaluation.

Full Analysis Set (FAS)

→

secondary · POSTED

Objective response rate was defined as the percentage of subjects whose best response was Complete Response (CR: disappearance of all clinical and radiological evidence of tumor (both target and non-target).) or Partial Response (PR: at least a 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum, no unequivocal progression of existing non-target lesions, and no appearance of new lesions.) according to Response Evaluation Criteria in Solid Tumors (RECIST v1.1). Results are based on central evaluation.

Full Analysis Set (FAS)

→

secondary · POSTED

Disease Control Rate (DCR) was defined as the percentage of subjects whose best response was Complete Response (CR: disappearance of all clinical and radiological evidence of tumor (both target and non-target).), Partial Response (PR: at least a 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum, no unequivocal progression of existing non-target lesions, and no appearance of new lesions.), or Stable Disease (SD: steady state of disease. Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, no unequivocal progression of existing non-target lesions, and no appearance of new lesions.) according to RECIST v1.1 criteria. SD had to be maintained for at least 12 weeks from the first demonstration of that rating. Results are based on central evaluation.

Full Analysis Set (FAS)

→

Reported safety

Serious events are not the same as severe events. Compare the original safety groups and their at-risk populations.

Open the study safety table →

Quality of life and patient-reported measures

No linked quality-of-life measure is recorded for this setting and study.

Full draft applicability and source-link record

trial

release id: clinical-indications-draft-1
disease state id: ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id: NCT01271712
trial label: Study of Regorafenib as a 3rd-line or Beyond Treatment for Gastrointestinal Stromal Tumors (GIST)
applicability: unclear
applicability note: Attached because the study's own record describes metastatic disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full.
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z
provenance class: regulator-cited
discovery note: Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
outcome Count: 6
harms Count: 5
qol Count: 0

outcomes

release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0000
source section id
NCT01271712-om-0000
source outcome index
0
endpoint
Progression-free Survival
endpoint definition
Progression-free Survival (PFS) was defined as the time from date of randomization to radiological disease progression or death due to any cause, whichever occurs first. PFS was based on central radiological assessment using modified RECIST (Response Evaluation Criteria in Solid Tumors) v.1.1. Progression is defined as at least a 20% increase in the sum of diameters of target lesions taking as reference the smallest sum on study; or unequivocal progression of existing non-target lesions; or appearance of new lesions. Subjects without progression or death at the time of analysis were censored at their last date of tumor evaluation. Results are based on central evaluation.
time origin
From randomization of the first subject until approximately 144 progression-free survival events had occurred (study duration approximately one year)
analysis role
primary
reporting status
POSTED
data cutoff
Source null
population note
Full Analysis Set (FAS) - defined as all randomized participants.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0001
source section id
NCT01271712-om-0001
source outcome index
1
endpoint
Overall Survival
endpoint definition
Overall Survival (OS) was defined as the time from date of randomization to death due to any cause. Subjects still alive at the time of analysis were censored at their date of last contact. Median OS was not observed at the time of PFS analysis and first analysis of OS, therefore only the proportion of death events was reported in the results posting system. This approach was maintained for the subsequent updates in the results posting system.
time origin
From randomization of the first subject until date of database cutoff (08 Jun 2015)
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Full Analysis Set (FAS). 58 (87.9%) patients in placebo group and 91 (68.4%) patients in regorafenib had started open-label treatment with regorafenib before time of final database cutoff 08 Jun 2015
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0002
source section id
NCT01271712-om-0002
source outcome index
2
endpoint
Time to Progression (TTP)
endpoint definition
Time to progression (TTP) was defined as the time from date of randomization to disease progression (based on central radiological assessment using modified RECIST \[Response Evaluation Criteria in Solid Tumors\] v.1.1). Progression is defined as at least a 20% increase in the sum of diameters of target lesions taking as reference the smallest sum on study; or unequivocal progression of existing non-target lesions; or appearance of new lesions. Subjects without progression at the time of analysis were censored at their last date of tumor evaluation. Results are based on central evaluation.
time origin
From randomization of the first subject until until date of database cutoff (26 Jan 2012); study duration approximately 1 year
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Full Analysis Set (FAS)
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0003
source section id
NCT01271712-om-0003
source outcome index
3
endpoint
Tumor Response
endpoint definition
Tumor Response of a subject was defined as the best tumor response (Complete Response \[CR: disappearance of all clinical and radiological evidence of tumor (both target and non-target).\], Partial Response \[PR: at least a 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum, no unequivocal progression of existing non-target lesions, and no appearance of new lesions.\], Stable Disease \[SD: steady state of disease. Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, no unequivocal progression of existing non-target lesions, and no appearance of new lesions.\], or Progressive Disease \[PD: at least a 20% increase in the sum of diameters of target lesions taking as reference the smallest sum on study or unequivocal progression of existing non-target lesions, or appearance of new lesions.\]) observed during the trial period and assessed according to RECIST v1.1 criteria. Results are based on central evaluation.
time origin
From randomization of the first subject until date of database cutoff (26 Jan 2012); study duration approximately 1 year
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Full Analysis Set (FAS)
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0004
source section id
NCT01271712-om-0004
source outcome index
4
endpoint
Objective Response Rate
endpoint definition
Objective response rate was defined as the percentage of subjects whose best response was Complete Response (CR: disappearance of all clinical and radiological evidence of tumor (both target and non-target).) or Partial Response (PR: at least a 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum, no unequivocal progression of existing non-target lesions, and no appearance of new lesions.) according to Response Evaluation Criteria in Solid Tumors (RECIST v1.1). Results are based on central evaluation.
time origin
From randomization of the first subject until date of database cutoff (26 Jan 2012); study duration approximately 1 year.
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Full Analysis Set (FAS)
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0005
source section id
NCT01271712-om-0005
source outcome index
5
endpoint
Disease Control Rate (DCR)
endpoint definition
Disease Control Rate (DCR) was defined as the percentage of subjects whose best response was Complete Response (CR: disappearance of all clinical and radiological evidence of tumor (both target and non-target).), Partial Response (PR: at least a 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum, no unequivocal progression of existing non-target lesions, and no appearance of new lesions.), or Stable Disease (SD: steady state of disease. Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, no unequivocal progression of existing non-target lesions, and no appearance of new lesions.) according to RECIST v1.1 criteria. SD had to be maintained for at least 12 weeks from the first demonstration of that rating. Results are based on central evaluation.
time origin
From randomization of the first subject until date of database cutoff (26 Jan 2012); study duration approximately 1 year
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Full Analysis Set (FAS)
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

arms

release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0000
arm key
OG000
arm label
Regorafenib (Stivarga, BAY73-4506)
regimen
Participants received Regorafenib (Stivarga) 160 mg (4 x 40 mg tablets) per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
133
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0000#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0000
arm key
OG001
arm label
Placebo
regimen
Participants received matching Placebo tablets per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
66
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0000#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0001
arm key
OG000
arm label
Regorafenib (Stivarga, BAY73-4506)
regimen
Participants received Regorafenib (Stivarga) 160 mg (4 x 40 mg tablets) per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
133
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0001#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0001
arm key
OG001
arm label
Placebo
regimen
Participants received matching Placebo tablets per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
66
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0001#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0002
arm key
OG000
arm label
Regorafenib (Stivarga, BAY73-4506)
regimen
Participants received Regorafenib (Stivarga) 160 mg (4 x 40 mg tablets) per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
133
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0002#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0002
arm key
OG001
arm label
Placebo
regimen
Participants received matching Placebo tablets per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
66
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0002#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0003
arm key
OG000
arm label
Regorafenib (Stivarga, BAY73-4506)
regimen
Participants received Regorafenib (Stivarga) 160 mg (4 x 40 mg tablets) per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
133
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0003#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0003
arm key
OG001
arm label
Placebo
regimen
Participants received matching Placebo tablets per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
66
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0003#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0004
arm key
OG000
arm label
Regorafenib (Stivarga, BAY73-4506)
regimen
Participants received Regorafenib (Stivarga) 160 mg (4 x 40 mg tablets) per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
133
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0004#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0004
arm key
OG001
arm label
Placebo
regimen
Participants received matching Placebo tablets per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
66
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0004#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0005
arm key
OG000
arm label
Regorafenib (Stivarga, BAY73-4506)
regimen
Participants received Regorafenib (Stivarga) 160 mg (4 x 40 mg tablets) per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
133
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0005#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
outcome key
om-0005
arm key
OG001
arm label
Placebo
regimen
Participants received matching Placebo tablets per os once daily, 3 weeks on therapy followed by 1 week off therapy to comprise a cycle of 4 weeks
analyzed count
66
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01271712-om-0005#groups/OG001

harms

release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
harm key
EG000
source section id
NCT01271712-ae
event group key
EG000
event group label
Regorafenib (Double Blind Only)
serious affected
23
serious at risk
41
other affected
40
other at risk
41
frequency threshold
5
collection period
From first administration of treatment till 30 days after last dose of treatment.
event definition
At primary completion (cutoff 26JAN2012) blinded patients who received either regorafenib or placebo were reported in "Regorafenib (DoubleBlindOnly)" and "Placebo (DoubleBlindOnly)" respectively; patients who received regorafenib after unblinding were reported in "Placebo, OpenLabelOnly(Switch to Regorafenib)". This safety update (cutoff 15APR2019) was reported in "Treated with Regorafenib at any time" and "Treated with Regorafenib for\>1 year".
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
harm key
EG001
source section id
NCT01271712-ae
event group key
EG001
event group label
Placebo (Double Blind Only)
serious affected
8
serious at risk
8
other affected
7
other at risk
8
frequency threshold
5
collection period
From first administration of treatment till 30 days after last dose of treatment.
event definition
At primary completion (cutoff 26JAN2012) blinded patients who received either regorafenib or placebo were reported in "Regorafenib (DoubleBlindOnly)" and "Placebo (DoubleBlindOnly)" respectively; patients who received regorafenib after unblinding were reported in "Placebo, OpenLabelOnly(Switch to Regorafenib)". This safety update (cutoff 15APR2019) was reported in "Treated with Regorafenib at any time" and "Treated with Regorafenib for\>1 year".
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
harm key
EG002
source section id
NCT01271712-ae
event group key
EG002
event group label
Placebo, Open Label Only (Switch to Regorafenib)
serious affected
31
serious at risk
58
other affected
58
other at risk
58
frequency threshold
5
collection period
From first administration of treatment till 30 days after last dose of treatment.
event definition
At primary completion (cutoff 26JAN2012) blinded patients who received either regorafenib or placebo were reported in "Regorafenib (DoubleBlindOnly)" and "Placebo (DoubleBlindOnly)" respectively; patients who received regorafenib after unblinding were reported in "Placebo, OpenLabelOnly(Switch to Regorafenib)". This safety update (cutoff 15APR2019) was reported in "Treated with Regorafenib at any time" and "Treated with Regorafenib for\>1 year".
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
harm key
EG003
source section id
NCT01271712-ae
event group key
EG003
event group label
Treated With Regorafenib at Any Time
serious affected
103
serious at risk
190
other affected
189
other at risk
190
frequency threshold
5
collection period
From first administration of treatment till 30 days after last dose of treatment.
event definition
At primary completion (cutoff 26JAN2012) blinded patients who received either regorafenib or placebo were reported in "Regorafenib (DoubleBlindOnly)" and "Placebo (DoubleBlindOnly)" respectively; patients who received regorafenib after unblinding were reported in "Placebo, OpenLabelOnly(Switch to Regorafenib)". This safety update (cutoff 15APR2019) was reported in "Treated with Regorafenib at any time" and "Treated with Regorafenib for\>1 year".
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-gastrointestinal-stromal-tumors-stivarga-dbeeb4f0
nct id
NCT01271712
harm key
EG004
source section id
NCT01271712-ae
event group key
EG004
event group label
Treated With Regorafenib for > 1 Year
serious affected
39
serious at risk
75
other affected
75
other at risk
75
frequency threshold
5
collection period
From first administration of treatment till 30 days after last dose of treatment.
event definition
At primary completion (cutoff 26JAN2012) blinded patients who received either regorafenib or placebo were reported in "Regorafenib (DoubleBlindOnly)" and "Placebo (DoubleBlindOnly)" respectively; patients who received regorafenib after unblinding were reported in "Placebo, OpenLabelOnly(Switch to Regorafenib)". This safety update (cutoff 15APR2019) was reported in "Treated with Regorafenib at any time" and "Treated with Regorafenib for\>1 year".
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

quality of life

setting source links

Preserved source evidence · Not medical advice