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Drugs in development · updated 30 October 2026

Gastrointestinal stromal tumor: drugs in development

Rare diseasePrevalence 1-5 / 10 000 according to Orphanet · ORPHA:44890

As of 30 October 2026 there are 40 active clinical trials (recruiting or ongoing, phases I–III) on 36 drugs for gastrointestinal stromal tumor. 17 are experimental, that is not yet approved in the United States.

40Active trials
17Experimental drugs
4Experimental, in phase III

Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.

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Experimental drugs

Not yet approved by the US FDA for any indication.

DrugMost advanced phaseActive trialsReference trial
VelzatinibIII3NCT07585266
Bezuclastinib TabletsCGT9486III2NCT05208047
IDRX-42III2NCT07218926
NB003II–III1NCT07379047
EnvafolimabKN035II2NCT07559864
ApatinibRivoceranibII1NCT05905887
BAY2927088II1NCT06760819
DCC-3009I–II1NCT06630234
InlexisertibI–II1NCT05957367
KQB198II1NCT07406633
LiporaxelII1NCT06326346
NSC #716051I–II1NCT01991379
PlixorafenibII1NCT05503797
RogaratinibII1NCT04595747
NN3201I1NCT06805825
OlverembatinibHQP1351I1NCT03594422
PimitespibTAS-116I1NCT05245968

Sorted by most advanced phase and number of active trials.

Approved drugs in new trials

Approved by the US FDA for at least one indication, not necessarily this disease, and now in active trials on it.

DrugMost advanced phaseActive trialsReference trial
ImatinibSTI571, Imkeldi®III12NCT07585266
SunitinibSutent®, Sunitinib Accord®III7NCT03673501
RegorafenibRegorafinib, BAY73-4506II–III5NCT07379047
RipretinibDCC-2618, Qinlock®III4NCT03673501
MidazolamMidazolam Hydrochloride (Autoinjector)®, Midazolam In 0.8% Sodium Chloride®III1NCT05208047
AldesleukinProleukinII1NCT07647068
AtezolizumabTecentriq, MPDL3280AII1NCT05152472
BinimetinibMEK162, Mektovi®I–II1NCT01991379
CyclophosphamideCyclophosphamid, CytoxanII1NCT07647068
FilgrastimRecombinant Human Granulocyte Colony-Stimulating Factor, NeupogenII1NCT07647068
FludarabineFludara, FLUDARABINII1NCT07647068
IpilimumabYERVOY, ipililumab IVII1NCT02834013
NivolumabOpdivo, ONO-4538II1NCT02834013
PaclitaxelTaxol, PaxlitaxelII1NCT05905887
PemigatinibPemazyre®II1NCT07434843

Sorted by most advanced phase and number of active trials. Showing 15 of 19 drugs.

All 36 drugs, with the details

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Broader categories

Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D046152 “Gastrointestinal Stromal Tumors”. Prevalence: Orphanet.

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