Gastrointestinal Stromal Tumor (GIST) Clinical Trials

Gastrointestinal stromal tumors (GISTs) originate from the interstitial cells of Cajal (ICC) or their precursors, cells that serve as pacemakers for gut motility. GISTs can arise anywhere in the GI tract but are most common in the stomach (60%) and small intestine (30%). They range from small, incidentally discovered tumors to large, aggressive malignancies.

The most common symptoms of Gastrointestinal Stromal Tumor include Abdominal pain or discomfort, A feeling of fullness after eating small amounts, Nausea and vomiting, Blood in stool or vomit (GI bleeding), Fatigue from chronic blood loss anemia, among others. About 4,000-6,000 new cases per year in the U.S.; true incidence may be higher as small GISTs are often incidentally discovered. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Gastrointestinal Stromal Tumor from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Mutational testing of your GIST is essential, as specific KIT or PDGFRA mutations predict response to different targeted therapies. Clinical trials are evaluating next-generation TKIs, combination strategies, and novel targets for TKI-resistant disease.

As of September 2026, Trial Friend's curated drug index does not include an FDA-approved treatment specific to Gastrointestinal Stromal Tumor. The Treatments section of this page checks FDA drug labels live and lists any approved drug whose label names the condition, and the trials listed below cover the investigational options. The FDA is due to decide on Bezuclastinib + sunitinib by November 30, 2026; the FDA calendar page carries the outcome the day it lands.

Gastrointestinal Stromal Tumor arises from acquired (somatic) genetic changes in the affected cells, commonly involving KIT, PDGFRA, SDHA, SDHB, SDHC and SDHD, rather than from an inherited variant. About 80% of GISTs carry a somatic KIT mutation and 10% PDGFRA; SDH-deficient GIST (under 10%) can reflect a germline SDHx variant, and rare familial KIT/PDGFRA GIST is autosomal dominant. About 4,000-6,000 new cases per year in the U.S.; true incidence may be higher as small GISTs are often incidentally discovered.

For Gastrointestinal Stromal Tumor the genetic testing that matters is usually of the tumor or affected blood cells rather than inherited DNA, looking for KIT, PDGFRA, SDHA, SDHB, SDHC and SDHD. About 80% of GISTs carry a somatic KIT mutation and 10% PDGFRA; SDH-deficient GIST (under 10%) can reflect a germline SDHx variant, and rare familial KIT/PDGFRA GIST is autosomal dominant.

Those results guide treatment choice and are often the entry requirement for targeted-therapy trials, so ask whether the biopsy was sent for molecular testing and request the report. The Genetic Testing section of this page lists laboratories in the NIH Genetic Testing Registry.

Eligibility for Gastrointestinal Stromal Tumor clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Gastrointestinal Stromal Tumor clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.