Clinical Trials Logo

Clinical Trial Summary

Objective: To follow people with GISTs and collect tumor tissue so that it can be studied in the lab. Eligibility: People age 6 and older who have a GIST. Design: Participants will be screened with a review of their medical records and samples. Participants will enroll in 1 other NIH study, and may be asked to enroll in 2 other optional NIH studies. Participants will have a medical history and physical exam. Data about how they function in their daily activities will be obtained. Participants may speak with a genetic counselor. They may have genetic testing. Participants will give blood samples. They may have a cheek swab. For this, small brush will be rubbed against the inside of the cheek. Participants may have a computed tomography (CT) scan of the chest, abdomen, and pelvis. Or they may have a CT scan of the chest and magnetic resonance imaging (MRI) of the abdomen and pelvis. Participants will be monitored every 6-12 months at the NIH Clinical Center, for up to 10 years before having surgery. If they need surgery, it will be performed at the NIH. Then, they will be monitored every 6-12 months, for up to 5 years after surgery. If a participant has surgery, tumor tissue samples will be taken. If a participant does not need surgery, their participation will end after 10 years. If they have surgery, the 5-year monitoring period will restart after each surgery.

Clinical Trial Description

Background: Gastrointestinal stromal tumors (GISTs) are the most common gastrointestinal soft tissue sarcoma, but remain a rare disease entity. Most GISTs are characterized by KIT or PDGFRA mutations, making them susceptible to tyrosine kinase inhibitor (TKI) therapy. Wild-type (WT) GISTs are rarer tumors, usually characterized by SDH mutations and/or lack of KIT or PDGFRA mutations; paragangliomas are frequently associated with WT GISTs. Non-WT GISTs may become refractory to TKI therapy, whereas WT GISTs are generally resistant to TKI therapy. The primary treatment modality for GISTs is surgical resection, which may involve the stomach, liver, and/or peritoneal surfaces; most patients will require multiple operations to remove disease not responsive to systemic agents. Investigational systemic therapies are limited by toxicity and/or lack of efficacy, resulting in an unmet need for novel treatment options. Obtaining fresh tumor tissue is critical to the successful development of GIST models for drug research, as well as for next generation tumor genomic sequencing, and to help identify novel targets and/or agents for the treatment of WT and TKI-resistant non-WT GISTs. Objective: Evaluate and follow patients with GISTs, particularly WT and treatment-refractory non-WT, to support translational research for this rare disease Assess disease-free intervals (DFIs) between surgical resection of disease for at least 5 years Eligibility: Participants with histologically confirmed or clinical presentation suspicious of GIST. Design: Prospective cohort study Participants with histologically confirmed or clinical presentation suspicious of GIST will enroll on study and will have active surveillance every 9-15 months for up to 10 years prior to and up to 5 years after surgical resection and/or cytoreduction. As participants may have multiple resections during the course of the study, 5-year surveillance post-surgery may be initiated multiple times, relative to the last resection performed. All participants enrolled will be evaluated for tumor resection or cytoreduction at the start of study and if appropriate, will be offered surgery, otherwise they will be on active surveillance until surgical resection or cytoreduction is clinically indicated. It is expected that approximately 30-40 patients per year may enroll on this trial; the accrual ceiling will be set at 400 to permit accrual over a 10-year period. ;

Study Design

Related Conditions & MeSH terms

NCT number NCT04557969
Study type Observational
Source National Institutes of Health Clinical Center (CC)
Contact Audra A Satterwhite, R.N.
Phone (240) 858-3552
Status Recruiting
Start date December 18, 2020
Completion date December 30, 2040

See also
  Status Clinical Trial Phase
Recruiting NCT05551416 - The EpiGASTRIC/EDGAR Project: New Strategies for the Early Detection and Prevention of Gastric Cancer
Recruiting NCT05518929 - Hypoxia During Gastroenterological Endoscope Procedures Sedated With Ciprofol In Overweight Or Obesity Patients Phase 4
Recruiting NCT06006390 - CEA Targeting Chimeric Antigen Receptor T Lymphocytes (CAR-T) in the Treatment of CEA Positive Advanced Solid Tumors Phase 1/Phase 2
Recruiting NCT03219593 - Apatinib as the First-Line Therapy in Elderly Locally Advanced or Metastatic Gastric Cancer Phase 2
Recruiting NCT05489211 - Study of Dato-Dxd as Monotherapy and in Combination With Anti-cancer Agents in Patients With Advanced Solid Tumours (TROPION-PanTumor03) Phase 2
Recruiting NCT05536102 - The Effectiveness and Safety of XELOX and Tislelizumab + PLD for Resectable Gastric Cancer (LidingStudy) Phase 2
Active, not recruiting NCT03170960 - Study of Cabozantinib in Combination With Atezolizumab to Subjects With Locally Advanced or Metastatic Solid Tumors Phase 1/Phase 2
Recruiting NCT06010862 - Clinical Study of CEA-targeted CAR-T Therapy for CEA-positive Advanced/Metastatic Malignant Solid Tumors Phase 1
Recruiting NCT05415098 - Study of Safety, Pharmacokinetic and Efficacy of APG-5918 in Advanced Solid Tumors or Lymphomas Phase 1
Active, not recruiting NCT04082364 - Combination Margetuximab, Retifanlimab, Tebotelimab, and Chemotherapy Phase 2/3 Trial in HER2+ Gastric/GEJ Cancer Phase 2/Phase 3
Withdrawn NCT03766607 - Trastuzumab Beyond Progression in HER2 Positive Metastatic Gastric Cancer Phase 2
Recruiting NCT04118114 - Phase II Study of PRL3-ZUMAB in Advanced Solid Tumors Phase 2
Completed NCT01924533 - Efficacy and Safety Study of Olaparib in Combination With Paclitaxel to Treat Advanced Gastric Cancer. Phase 3
Terminated NCT01641939 - A Study of Trastuzumab Emtansine Versus Taxane in Participants With Human Epidermal Growth Factor Receptor 2 (HER2)-Positive Advanced Gastric Cancer Phase 2/Phase 3
Recruiting NCT05107674 - A Study of NX-1607 in Adults With Advanced Malignancies Phase 1
Active, not recruiting NCT04908813 - Study of HLX22 in Combanition With Trastuzumab and Chemotherapy Versus Placebo in Combination With Trastuzumab and Chemotherapy for Treatment of Locally Advanced or Metastatic Gastric Cancer Phase 2
Active, not recruiting NCT04249739 - Pembrolizumab + Capecitabine´╝ĆOxaliplatin (CapeOx) -HER2 Nagative and Pembrolizumab + Trastuzumab + Cisplatin/Capecitabine HER2 Positive Phase 2
Recruiting NCT05514158 - To Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Disitamab Vedotin Combined With RC98 in the Treatment of Subjects With HER2-expressing Locally Advanced or Metastatic Gastric Cancer (Including AEG) Phase 1
Recruiting NCT04931654 - A Study to Assess the Safety and Efficacy of AZD7789 in Participants With Advanced or Metastatic Solid Cancer Phase 1/Phase 2
Recruiting NCT03175224 - APL-101 Study of Subjects With NSCLC With c-Met EXON 14 Skip Mutations and c-Met Dysregulation Advanced Solid Tumors Phase 2