DrugEvidence

Diseases / Gastrointestinal Stromal Tumors

Gastrointestinal Stromal Tumors

Disease

Gastrointestinal stromal tumors (GISTs) are rare mesenchymal neoplasms of the gastrointestinal tract, commonly driven by activating KIT or PDGFRA mutations and arising from interstitial cells of Cajal. They can occur throughout the GI tract, most often in the stomach and small intestine. Clinical behavior ranges from indolent to malignant, and management depends on tumor risk features and molecular profile.

Trials
333
Recruiting
46
Completed
178
With results
69

Last revised

Pipeline

Phase by status

PhaseRecruitingNot yetBy invitationActiveCompletedTerminatedWithdrawnSuspendedUnknownApproved for marketingAvailableNo longer availableTotal
Early Phase 1 11 2
Phase 1 52294111 43
Phase 1/2 211421 20
Phase 2 16215669210 111
Phase 2/3 11 2
Phase 3 3121893 36
Phase 4 82 10
Not applicable 5195111 32
Unknown 14413223112125 77

Trials started per year

2021
4
2022
17
2023
10
2024
10
2025
15
2026
12
2027
1

Conditions studied, by phase

ConditionEarly Phase 1Phase 1Phase 1/2Phase 2Phase 2/3Phase 3Phase 4Not applicableUnknownTotal
Gastrointestinal Stromal Tumors 24320111236103277 333
Sarcoma 8716214 38
Stomach Neoplasms 9461179 37
Pancreatic Neoplasms 7331157 27
Esophageal Neoplasms 7121137 22
Colonic Neoplasms 6321126 21
Colorectal Neoplasms 544152 21
Gastrointestinal Neoplasms 1262127 21
Neoplasm Metastasis 335316 21
Neoplasms 7262121 21

Most active sponsors

#SponsorTrialsPhase 3
1Novartis Pharmaceuticals254
2National Cancer Institute (NCI)233
3Asan Medical Center182
4Deciphera Pharmaceuticals, LLC93
5Pfizer92
6European Organisation for Research and Treatment of Cancer - EORTC73
7M.D. Anderson Cancer Center70
8Bayer61
9Blueprint Medicines Corporation51
10First Affiliated Hospital, Sun Yat-Sen University50

Most studied interventions

#InterventionTrials
1Imatinib Mesylate71
2Sunitinib31
3regorafenib17
4nilotinib15
5ripretinib13
6avapritinib7
7Dasatinib5
8Ipilimumab5
9Laparoscopy5
104-amino-5-fluoro-3-(5-(4-methylpiperazin-1-yl)-1H-benzimidazol-2-yl)quinolin-2(1H)-one4

Evidence

Results reported
38.8%69 of 178 completed
Published
13.5%24 of 178 completed
Primary endpoint met
75%6 of 8 analyses
Median months to report
14

Documents disclosed

Protocol available
27
Statistical analysis plan
25
PubMed citations
147

Trial profile

Median enrollment
57
Median duration
47months
Countries
58
Study sites
3298

Who is enrolled

Adults
333
Children
43
Older adults
322
Healthy volunteers
8

Median enrollment by phase

Early Phase 1
18
Phase 1
34
Phase 1/2
68
Phase 2
40
Phase 2/3
152
Phase 3
133
Phase 4
56
Not applicable
61
Unknown
100

Where trials run

United States
137
China
65
France
47
South Korea
41
Germany
40
Italy
32
Spain
30
Canada
28
Netherlands
28
United Kingdom
25

Busiest cities

#CityCountryTrials
1BostonUnited States54
2New YorkUnited States42
3PhiladelphiaUnited States42
4SeoulSouth Korea39
5HoustonUnited States37
6PortlandUnited States36
7LyonFrance35
8BeijingChina31
9BordeauxFrance29
10Los AngelesUnited States27

Terminations

Terminated
31
Withdrawn
7
Suspended
3
Stopped for recruitment
4.2%14 of 333 trials

Stated reasons

Other / unclear
15
Recruitment
14
Business decision
4
Lack of efficacy
3
Regulatory
2
COVID-19
1
Funding
1
Safety
1

Reasons are grouped from the sponsor's own wording with a rule-based classifier; the original text is shown below.

Recently stopped

TrialStatusStated reasonStopped
NCT05867901 Suspended Suspended Enrollment has been temporarily paused pending ethics review and implementation of the revised study protocol. Follow-up of previously enrolled participants is continuing. 2028-12-31
NCT05080621 Withdrawn Withdrawn The Sponsor decided to discontinue this study due to a corporate restructuring intended to prioritize clinical development of select programs. No patients enrolled in this study and no patients received investigational product. 2027-05-31
NCT04714983 Suspended Suspended DNAtrix management decision not based on study design, safety, or site performance. 2026-12-31
NCT02734823 Withdrawn Withdrawn Lack of accrual 2024-03-14
NCT05160168 Terminated Terminated The Sponsor terminated the study due to early dose-limiting toxicities. 2024-02-02
NCT04000529 Terminated Terminated Business reasons 2024-01-15
NCT04811833 Withdrawn Withdrawn The GASTROMO study has been stopped early before enrolling its first patient because the results of our primary endpoint would have been inconsistent. The variability of the leakage rate between all anastomosis included in the study was overly high. 2023-12-31
NCT02924714 Suspended Suspended Slow accrual 2022-11-30
NCT04879979 Terminated Terminated Slow enrollment 2022-05-04
NCT04409223 Terminated Terminated R\&D strategy adjustment 2022-03-25

Trial records