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

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ClinicalTrials.gov Identifier: NCT04557969
Recruitment Status : Not yet recruiting
First Posted : September 22, 2020
Last Update Posted : October 23, 2020
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

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 3 other 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.

...


Condition or disease
Gastric Cancer Gastric Neoplasm Gastrointestinal Stromal Sarcoma Gastrointestinal Stromal Neoplasm Gastrointestinal Stromal Tumor (GIST)

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 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:

-Patients with histologically confirmed or clinical presentation suspicious of GIST.

Design:

  • Prospective cohort study
  • Patients with histologically confirmed or clinical presentation suspicious of GIST will enroll on study and will have active surveillance every 6 months for up to 10 years prior to and up to 5 years after surgical resection and/or cytoreduction. As patients 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 patients 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.

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Study Type : Observational
Estimated Enrollment : 400 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Prospective Study of Surgery in Gastrointestinal Stromal Tumors (GISTs) for Treatment, Tumor Modeling, and Genomic Analysis
Estimated Study Start Date : October 28, 2020
Estimated Primary Completion Date : December 30, 2040
Estimated Study Completion Date : December 30, 2040


Group/Cohort
1/ Cohort 1
Patients with histologically confirmed or clinical presentation suspicious of GIST



Primary Outcome Measures :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. Characterize genomic and clinicopathologic features of GISTs [ Time Frame: at clinical visits and follow-up ]
    Characterization of the genomic and clinicopathologic features associated with GISTs.



Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Individuals with histologically confirmed or clinical presentation suspicious of GIST.
Criteria
  • INCLUSION CRITERIA:
  • 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.
  • Ability and willingness to enroll on 13C0176, 'Tumor, Normal Tissue and Specimens from Patients Undergoing Evaluation or Surgical Resection of Solid Tumors'.
  • Ability and willingness to enroll on 06C0213, 'Tissue Procurement Protocol'.
  • Ability and willingness to enroll on 19C0016, 'Natural History and Biospecimen Acquisition Study for Children and Adults with Rare Solid Tumors'.

EXCLUSION CRITERIA:

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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04557969


Contacts
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Contact: Cathleen E Hannah (240) 409-8860 cathleen.hannah@nih.gov

Locations
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United States, Maryland
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office    888-624-1937      
Sponsors and Collaborators
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Andrew M Blakely, M.D. National Cancer Institute (NCI)
Additional Information:
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Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT04557969    
Other Study ID Numbers: 200161
20-C-0161
First Posted: September 22, 2020    Key Record Dates
Last Update Posted: October 23, 2020
Last Verified: September 15, 2020
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):
Tyrosine Kinase Inhibitor (TKI) Therapy
Wild-Type GISTs (WT GISTs)
PDGFRA Mutation
KIT Mutation
SDH Mutation
Additional relevant MeSH terms:
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Neoplasms
Stomach Neoplasms
Gastrointestinal Stromal Tumors
Sarcoma, Endometrial Stromal
Sarcoma
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Gastrointestinal Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Gastrointestinal Diseases
Stomach Diseases
Neoplasms, Connective Tissue
Neoplasms, Complex and Mixed
Endometrial Stromal Tumors
Endometrial Neoplasms
Uterine Neoplasms
Uterine Diseases
Genital Neoplasms, Female
Urogenital Neoplasms