Adults 3 to 99, any sex, with GIST. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With a Clinical Activity-radiographic ResponsePrimary· Every 3 cycles x4 and then every 6 cycles (1 cycle = 28 days) until removal from protocol therapy, an average of 12 months.
Clinical activity will be assessed primarily by radiographic response of measurable disease using the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Complete Response is disappearance of all target lesions. Any pathological lymph nodes must have reduction in short axis to \<10 mm. Partial Response is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Progressive Disease is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baselin
Group
Value
95% CI
200 mg Vandetanib Adult
0
300 mg Vandetanib Adult
0
Dose Level 100mg/m^2 Vandetanib Pediatric
0
200 mg Vandetanib Adult
0
300 mg Vandetanib Adult
0
Dose Level 100mg/m^2 Vandetanib Pediatric
0
200 mg Vandetanib Adult
1
300 mg Vandetanib Adult
2
Dose Level 100mg/m^2 Vandetanib Pediatric
1
200 mg Vandetanib Adult
1
300 mg Vandetanib Adult
3
Dose Level 100mg/m^2 Vandetanib Pediatric
1
Count of Participants With Serious and Non-serious Adverse EventsSecondary· Date treatment consent signed to date off study, approximately 32 months and 1 day.
The count of participants with serious and non-serious adverse events was assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of th
Group
Value
95% CI
200 mg Vandetanib Adult
2
300 mg Vandetanib Adult
5
Dose Level 100mg/m^2 Vandetanib Pediatric
2
Percentage of Participants Overall SurvivalSecondary· Overall survival was computed using the number of months from the date of on study to the date of death, an average of 12 months.
Overall survival is defined as the date of on-study to the date of death from any cause or last follow up.
Group
Value
95% CI
200 mg Vandetanib Adult
50
0.6 – 91
300 mg Vandetanib Adult
60
12.6 – 88.2
Dose Level 100mg/m^2 Vandetanib Pediatric
50
0.6 – 91
Progression Free-SurvivalSecondary· Patients will be evaluated approximately 60 days after last dose of investigational drug until removal of protocol therapy, an average of 12 months.
Progression free survival is defined as the time interval from start of treatment to documented evidence of disease progression. Disease progression was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 and is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. (Note: the appearance of one or more new lesions is also considered
Group
Value
95% CI
200 mg Vandetanib Adult
4
0.6 – 91
300 mg Vandetanib Adult
5.1
1.8 – 22.5
Dose Level 100mg/m^2 Vandetanib Pediatric
13.4
2.7 – 24.1
Maximum Standardized Uptake Value (SUVmax) on Fluorodeoxyglucose Positron Emission Tomography (FDG-PET)Secondary· Baseline and at a subsequent PET performed on or about day 3-6 of cycle 1
The maximum standardized uptake value (SUVmax) was used to measure the uptake of FDG-PET by the Gastrointestinal Tumors.
Baseline
Group
Value
95% CI
200 mg Vandetanib Adult
22.4
22.4 – 22.4
300 mg Vandetanib Adult
21.4
15.6 – 30.7
Day 3-6 of cycle 1
Group
Value
95% CI
200 mg Vandetanib Adult
22.0
22.0 – 22.0
300 mg Vandetanib Adult
12.4
7.5 – 19.3
Adverse events — posted to ClinicalTrials.gov
Time frame: Date treatment consent signed to date off study, approximately 32 months and 1 day..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Background:
-Some people with wild-type gastrointestinal stromal tumors (WT-GIST) have a deficiency in one of their proteins called succinate dehydrogenase (SDH). Vandetanib is a cancer drug that has been approved to treat thyroid cancer and has been used with some success in other tumors that have a similar loss of SDH. Researchers want to see if this drug can also decrease tumor growth in people with WT-GIST.
Objectives:
-To test whether the study drug will benefit people with WT-GIST.
Eligibility:
-Adults and children 3 years old and older with WT-GIST.
Design:
* Researchers will test participants tumor tissue to confirm it is the wild type of GIST.
* Participants will be screened with a medical history, physical exam, and blood tests. They will also have electrical recording of the heart (Eastern Cooperative Oncology Group (ECOG)) and scans of the tumor.
* Participants will take the study drug in 28-day cycles. Their doctor will decide how many cycles they can complete.
* They will take the study drug once every day and record it in a diary.
* On Day 14, they will also visit their doctor to look for side effects.
* Before cycles 2, 3 and 4, participants will have a physical exam, urine tests, blood pressure check, and blood tests. These tests will then be done periodically for as long as they are in the study.
* Before cycle 4, scans will be done to check the size of the cancer. Most of these will be repeated every 3-6 cycles.
* When they stop taking the study drug, participants will return to the clinic for a physical exam and blood tests.
Publications & conference data
7 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04760288 — A Study of Pralsetinib Versus Standard of Care (SOC) for Treatment of RET-Mutated Medullary Thyroid Cancer (MTC).
· Phase 3
· withdrawn
NCT06482086 — Efficacy of Organoid-Based Drug Screening to Guide Treatment for Locally Advanced Thyroid Cancer
· Phase 2
· recruiting
NCT03630120 — Adaptive Tyrosine Kinase Inhibitor (TKI) Therapy In Patients With Thyroid Cancer
· Phase 2
· terminated
NCT02495103 — Vandetanib in Combination With Metformin in People With HLRCC or SDH-Associated Kidney Cancer or Sporadic Papillary Rena
· Phase 1, PHASE2
· terminated
NCT01823068 — Vandetanib in Advanced NSCLC With RET Rearrangement
· Phase 2
· completed
Other recruiting trials for GIST
Currently open trials in the same condition.
NCT07096609 — Lenvatinib After Progression on Imatinib, Sunitinib, and Regorafenib for GIST Patients
· Phase 1, PHASE2
· recruiting
NCT07411118 — RCT of EFTR Versus STER for GIST Treatment
· NA
· recruiting
NCT06640361 — A Study of Olverembatinib in SDH-deficient GIST.
· Phase 3
· recruiting
NCT05804331 — The Australia and New Zealand Multicentre Upper Gastrointestinal Endoscopic Tissue Resection Study
· recruiting
NCT03050268 — Familial Investigations of Childhood Cancer Predisposition
· recruiting
Other National Cancer Institute (NCI) trials
Trials by the same sponsor.
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· Phase 1, PHASE2
· recruiting
NCT07572123 — Evaluating the Addition of Maintenance Immunotherapy Compared to the Usual Treatment of Chemotherapy and Autologous Stem
· Phase 2, PHASE3
· not yet recruiting
NCT07281417 — Testing the Addition of Cemiplimab (REGN2810) to Chemotherapy Treatment Given Prior to Surgery in Patients With Sinonasa
· Phase 2
· recruiting
NCT07012044 — A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After
· Phase 1
· not yet recruiting
NCT07437950 — Comparing Different Treatment Lengths for Venetoclax in Older People With Newly Diagnosed Acute Myeloid Leukemia (A Myel
· Phase 2
· not yet recruiting
Publications: Europe PMC API search by NCT ID, retrieved 9 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Cancer Institute (NCI)
Last refreshed: 30 March 2020
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02015065.