Ignite Creation Date:
2025-12-24 @ 10:55 PM
Ignite Modification Date:
2026-01-02 @ 1:06 AM
Study NCT ID:
NCT04557969
Status:
RECRUITING
Last Update Posted:
2025-11-28
First Post:
2020-09-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Surgery in Gastrointestinal Stromal Tumors (GISTs) for Treatment, Tumor Modeling, and Genomic Analysis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013274', 'term': 'Stomach Neoplasms'}, {'id': 'D046152', 'term': 'Gastrointestinal Stromal Tumors'}], 'ancestors': [{'id': 'D005770', 'term': 'Gastrointestinal Neoplasms'}, {'id': 'D004067', 'term': 'Digestive System Neoplasms'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D005767', 'term': 'Gastrointestinal Diseases'}, {'id': 'D013272', 'term': 'Stomach Diseases'}, {'id': 'D009372', 'term': 'Neoplasms, Connective Tissue'}, {'id': 'D018204', 'term': 'Neoplasms, Connective and Soft Tissue'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 400}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2020-12-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09-10', 'completionDateStruct': {'date': '2040-12-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-26', 'studyFirstSubmitDate': '2020-09-18', 'studyFirstSubmitQcDate': '2020-09-18', 'lastUpdatePostDateStruct': {'date': '2025-11-28', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2020-09-22', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2040-12-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Evaluate and follow patients with GISTs, particularly WT or treatment-refractory non-WT, to support translational research for this rare disease', 'timeFrame': 'on-going', 'description': '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.'}, {'measure': 'Assess disease-free intervals (DFIs) between surgical resection of disease for at least 5 years', 'timeFrame': 'surveillance every 6-12 months and time of surgery, until 5 years after last surgical resection (relative to the last resection performed)', 'description': '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.'}], 'secondaryOutcomes': [{'measure': 'Characterize genomic and clinicopathologic features of GISTs', 'timeFrame': 'at clinical visits and follow-up', 'description': 'Characterization of the genomic and clinicopathologic features associated with GISTs.'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Tyrosine Kinase Inhibitor (TKI) Therapy', 'Wild-Type GISTs (WT GISTs)', 'PDGFRA Mutation', 'KIT Mutation', 'SDH Mutation', 'Natural History'], 'conditions': ['Gastric Cancer', 'Gastric Neoplasm', 'Gastrointestinal Stromal Sarcoma', 'Gastrointestinal Stromal Neoplasm', 'Gastrointestinal Stromal Tumor (GIST)']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_2020-C-0161.html', 'label': 'NIH Clinical Center Detailed Web Page'}]}, 'descriptionModule': {'briefSummary': 'Objective:\n\nTo follow people with GISTs and collect tumor tissue so that it can be studied in the lab.\n\nEligibility:\n\nPeople age 6 and older who have a GIST.\n\nDesign:\n\nParticipants will be screened with a review of their medical records and samples.\n\nParticipants will enroll in 1 other NIH study, and may be asked to enroll in 2 other optional NIH studies.\n\nParticipants will have a medical history and physical exam. Data about how they function in their daily activities will be obtained.\n\nParticipants may speak with a genetic counselor. They may have genetic testing.\n\nParticipants will give blood samples. They may have a cheek swab. For this, small brush will be rubbed against the inside of the cheek.\n\nParticipants 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.\n\nParticipants 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.\n\nIf a participant has surgery, tumor tissue samples and research specimen will be taken.\n\nIf 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.', 'detailedDescription': 'Background:\n\nGastrointestinal stromal tumors (GISTs) are the most common gastrointestinal soft tissue sarcoma, but remain a rare disease entity.\n\nMost GISTs are characterized by KIT or PDGFRA mutations, making them susceptible to tyrosine kinase inhibitor (TKI) therapy.\n\nWild-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.\n\nNon-WT GISTs may become refractory to TKI therapy, whereas WT GISTs are generally resistant to TKI therapy.\n\nThe 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.\n\nInvestigational systemic therapies are limited by toxicity and/or lack of efficacy, resulting in an unmet need for novel treatment options.\n\nObtaining 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.\n\nObjective:\n\nEvaluate and follow participants with GISTs, particularly WT and treatment-refractory non-WT, to support translational research for this rare disease\n\nAssess disease-free intervals (DFIs) between surgical resection of disease for at least 5 years\n\nEligibility:\n\nParticipants with histologically confirmed or clinical presentation suspicious of GIST.\n\nDesign:\n\nProspective cohort study\n\nParticipants 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.\n\nAll 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.\n\nIt 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Individuals with histologically confirmed or clinical presentation suspicious of GIST.', 'healthyVolunteers': False, 'eligibilityCriteria': '* INCLUSION CRITERIA:\n* 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.\n* Age \\>= 6 years\n* ECOG performance status \\<= 2 (Karnofsky or Lansky \\>= 60%)\n* Ability of participant or parent/guardian to understand and the willingness to sign a written informed consent document.\n\nEXCLUSION CRITERIA:\n\n\\- Non-modifiable medical comorbidities that would preclude cytoreductive surgery.'}, 'identificationModule': {'nctId': 'NCT04557969', 'briefTitle': 'Surgery in Gastrointestinal Stromal Tumors (GISTs) for Treatment, Tumor Modeling, and Genomic Analysis', 'organization': {'class': 'NIH', 'fullName': 'National Institutes of Health Clinical Center (CC)'}, 'officialTitle': 'Prospective Study of Surgery in Gastrointestinal Stromal Tumors (GISTs) for Treatment, Tumor Modeling, and Genomic Analysis', 'orgStudyIdInfo': {'id': '200161'}, 'secondaryIdInfos': [{'id': '20-C-0161'}]}, 'armsInterventionsModule': {'armGroups': [{'label': '1/ Cohort 1', 'description': 'Patients with histologically confirmed or clinical presentation suspicious of GIST'}]}, 'contactsLocationsModule': {'locations': [{'zip': '20892', 'city': 'Bethesda', 'state': 'Maryland', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'For more information at the NIH Clinical Center contact National Cancer Institute Referral Office', 'role': 'CONTACT', 'phone': '888-624-1937'}], 'facility': 'National Institutes of Health Clinical Center', 'geoPoint': {'lat': 38.98067, 'lon': -77.10026}}], 'centralContacts': [{'name': 'Stephanie N Canady, R.N.', 'role': 'CONTACT', 'email': 'stephanie.canady@nih.gov', 'phone': '(240) 858-7573'}, {'name': 'Andrew M Blakely, M.D.', 'role': 'CONTACT', 'email': 'andrew.blakely@nih.gov', 'phone': '(240) 760-7647'}], 'overallOfficials': [{'name': 'Andrew M Blakely, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'National Cancer Institute (NCI)'}]}, 'ipdSharingStatementModule': {'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'ICF'], 'timeFrame': '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.', 'ipdSharing': 'YES', 'description': '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.', 'accessCriteria': '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.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'National Cancer Institute (NCI)', 'class': 'NIH'}, 'responsibleParty': {'type': 'SPONSOR'}}}}