Surgery in Gastrointestinal Stromal Tumors (GISTs) for Treatment, Tumor Modeling, and Genomic Analysis

NCT04557969 | Recruiting

• 2 other identifiers: 20016120-C-0161
• Study Type: observational
• Target: 400
• Locations: 1 country
• Sites: 1

Brief 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 and research specimen 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.

Conditions

Gastric Neoplasm; Gastrointestinal Stromal Sarcoma; Gastric Cancer; Gastrointestinal Stromal Neoplasm; Gastrointestinal Stromal Tumor (GIST)

Keywords

Tyrosine Kinase Inhibitor (TKI) Therapy; Wild-Type GISTs (WT GISTs); PDGFRA Mutation; KIT Mutation; SDH Mutation; Natural History

Outcome Measures

Primary Outcomes (2)

• Evaluate and follow patients with GISTs, particularly WT or treatment-refractory non-WT, to support translational research for this rare disease

  Patients with non-WT GIST and WT GIST will have the durations of the DFIs described both within each patient as their own control and across patients. Analyses will be done separately for those with WT GIST and those with non-WT GIST as well as for all patients combined.

  on-going

• Assess disease-free intervals (DFIs) between surgical resection of disease for at least 5 years

  Surveillance every 6-12 months and time of surgery, by tumor measurement and imaging studies, to assess disease-free intervals (DFIs) between surgical resection of disease for at least 5 years.

  surveillance every 6-12 months and time of surgery, until 5 years after last surgical resection (relative to the last resection performed)

Secondary Outcomes (1)

• Characterize genomic and clinicopathologic features of GISTs

  at clinical visits and follow-up

Study Arms (1)

1/ Cohort 1

Patients with histologically confirmed or clinical presentation suspicious of GIST

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Individuals with histologically confirmed or clinical presentation suspicious of GIST.

You may qualify if:

• Histological confirmation or clinical presentation suspicious of GIST; histological confirmation will be preferably by review of archival tissue if available, fresh biopsy will not be required if inadequate tissue sample.
• Age \>= 6 years
• ECOG performance status \<= 2 (Karnofsky or Lansky \>= 60%)
• Ability of participant or parent/guardian to understand and the willingness to sign a written informed consent document.

You may not qualify if:

• \- Non-modifiable medical comorbidities that would preclude cytoreductive surgery.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Stomach Neoplasms; Gastrointestinal Stromal Tumors

Condition Hierarchy (Ancestors)

Gastrointestinal Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Stomach Diseases; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type

Study Officials

• Andrew M Blakely, M.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Stephanie N Canady, R.N.

CONTACT

(240) 858-7573; stephanie.canady@nih.gov

Andrew M Blakely, M.D.

CONTACT

(240) 760-7647; andrew.blakely@nih.gov

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 18, 2020
• First Posted: September 22, 2020
• Study Start: December 18, 2020
• Primary Completion (Estimated): December 30, 2040
• Study Completion (Estimated): December 30, 2040
• Last Updated: May 1, 2026

Record last verified: 2026-02-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Clinical data available during the study and indefinitely.@@@@@@Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active.
• Access Criteria: Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.@@@@@@Genomic data are made available via dbGaP through requests to the data custodians.

Locations

US (1)