Gastric CancerNCT04557969
Surgery in Gastrointestinal Stromal Tumors (GISTs) for Treatment, Tumor Modeling, and Genomic Analysis
An observational study of Gastric Cancer and Gastric Neoplasm, run by National Cancer Institute (NCI) at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Gastric Cancer, Gastric Neoplasm, Gastrointestinal Stromal Sarcoma
- How many people
- 400 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Dec 2020to 30 Dec 2040
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals with histologically confirmed or clinical presentation suspicious of GIST.
Who may be able to join
- 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.
Who cannot join
- Non-modifiable medical comorbidities that would preclude cytoreductive surgery.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
For more information at the NIH Clinical Center contact National Cancer Institute Referral Office, 888-624-1937
About this study
Official title: Prospective Study of Surgery in Gastrointestinal Stromal Tumors (GISTs) for Treatment, Tumor Modeling, and Genomic Analysis
Objective:
To follow people with GISTs and collect tumor tissue so that it can be studied in the lab.
Read more
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.
...
Read the detailed 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 participants 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 participants per year may enroll on this trial; the accrual ceiling will be set at 400 to permit accrual over a 10-year period.
- Conditions
- Gastric Cancer, Gastric Neoplasm, Gastrointestinal Stromal Sarcoma, 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
- Medical subject headings
- Stomach Neoplasms, Gastrointestinal Stromal Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 1/ Cohort 1 | Patients with histologically confirmed or clinical presentation suspicious of GIST |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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
Characterization of the genomic and clinicopathologic features associated with GISTs.
Measured: at clinical visits and follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2020
- Study start18 Dec 2020
- Primary completion30 Dec 2040 (estimated)
- Study completion30 Dec 2040 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gastric CancerStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 400 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Andrew M Blakely, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04557969
- Sponsor's protocol ID
- 200161
- Other IDs
- 20-C-0161
- Status last verified
- 13 Oct 2026
- First submitted
- 18 Sep 2020
- Last updated
- 29 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: All IPD recorded in the medical record will be shared with intramural investigators upon request. @@@@@@In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Study of Surgery in Gastrointestinal Stromal Tumors (GISTs) for Treatment, Tumor Modeling, and Genomic Analysis
- ClinicalTrials.gov ID
- NCT04557969
- Acronym / sponsor ID
- 200161
- Conditions
- Gastric Cancer, Gastric Neoplasm, Gastrointestinal Stromal Sarcoma, 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
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 400 participants (estimated)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 18 Dec 2020 (actual)
- Primary completion
- 30 Dec 2040 (estimated)
- Study completion
- 30 Dec 2040 (estimated)
- First posted
- 22 Sep 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
...
Detailed 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 participants 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 participants 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
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1/ Cohort 1 | – | –Patients with histologically confirmed or clinical presentation suspicious of GIST |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals with histologically confirmed or clinical presentation suspicious of GIST.
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (1 listed)
- Non-modifiable medical comorbidities that would preclude cytoreductive surgery.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Evaluate and follow patients with GISTs, particularly WT or treatment-refractory non-WT, to support translational research for this rare disease
Time frame: on-going
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.
Assess disease-free intervals (DFIs) between surgical resection of disease for at least 5 years
Time frame: surveillance every 6-12 months and time of surgery, until 5 years after last surgical resection (relative to the last resection performed)
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.
1 secondary outcome
Characterize genomic and clinicopathologic features of GISTs
Time frame: at clinical visits and follow-up
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. All IPD recorded in the medical record will be shared with intramural investigators upon request. @@@@@@In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.
Related trials for Gastric Cancer
RecruitingPhase 1/2
Study of INBRX-106 and INBRX-106 in Combination With Pembrolizumab (Keytruda®) in Subjects With Locally Advanced or Metastatic Solid Tumors (Hexavalent OX40 Agonist)
Solid Tumor, Non-Small Cell Lung Cancer
RecruitingPhase 2/3
Multicenter Validation Trial of [18F]AlF-FAPI-74 for PET Imaging of Cancer-associated Fibroblasts Through Fibroblast Activation Protein Inhibitors (FAPI) in Different Tumor Types
Oesophageal Cancer, Gastric Cancer
Not yet recruitingPhase 1
GK01 Cell Therapy in Gastric Cancer With Peritoneal Metastasis
Gastric Cancer (GC)
RecruitingPhase 3
A Study of Neoadjuvant-adjuvant Durvalumab and FLOT Chemotherapy Followed by Adjuvant Durvalumab in Chinese Patients With Resectable GC/GEJC.
Gastric, Esophagogastric Junction
Active, not recruitingEarly Phase 1
Iomab-ACT: A Pilot Study of 131-I Apamistamab Followed by CD19-Targeted CAR T-Cell Therapy for Patients With Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia or Diffuse Large B-Cell Lymphoma
B-ALL, DLBCL
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Andrew M Blakely, M.D. principal investigator
National Cancer Institute (NCI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Gastric Cancer and Gastric Neoplasm, run by National Cancer Institute (NCI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Stephanie N Canady, R.N. ((240) 858-7573, stephanie.canady@nih.gov), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate and follow patients with GISTs, particularly WT or treatment-refractory non-WT, to support translational research for this rare disease (time frame: on-going). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Oct 2026.