Gastrointestinal Stromal TumorsPhase 2NCT05366816
ctDNA-Guided Sunitinib And Regorafenib Therapy for GIST
A Phase 2 study testing Sunitinib and Regorafenib in people with Gastrointestinal Stromal Tumors, run by University of Miami at 1 site in United States.
Trial summary
The purpose of this research is to test if mutations (changes in DNA) in exons (segment of DNA or RNA containing information that has the instructions for making proteins) in the KIT gene can be used to predict the body's response to standard of care treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gastrointestinal Stromal Tumors
- What is being tested?
- Sunitinib and Regorafenib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Oct 2023to 31 Dec 2028
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients who are ≥ 18 years of age.
- Patients who have histologically confirmed metastatic or unresectable GIST. Unresectable GIST must be confirmed to be unresectable by an experienced surgeon.
- Patients may have had any prior lines of TKI therapy.
- Patients who received imatinib prior treatment regimens, including adjuvant therapy, with objective disease progression, inadequate clinical benefit, or intolerance. Additionally, disease progression or intolerance to other systemic therapies including investigational agents and radiotherapy is allowed. --- Patients who experienced intolerance to prior therapies must have objective disease progression prior to enrollment.
- Patients who have an ECOG PS of 0 to 2.
- Patient, or legal guardian if permitted by local regulatory authorities, who provides informed consent to participate in the study.
- Presence of KIT exon 13 or 17 secondary mutation will be determined through a circulating tumor DNA (ctDNA) blood test or biopsy performed as per standard of care.
Who cannot join
- Patients who have received prior treatment with sunitinib or regorafenib.
- Patients who have received more than 2 different prior tyrosine kinase inhibitor (TKI) treatment regimens. If a patient receives the same TKI more than once sequentially (eg, imatinib followed by a period without systemic therapy and retreatment with imatinib), that will be counted as a single TKI treatment regimen.
- Patients who are known to be KIT wild type.
- Patients who received any systemic anticancer therapy within 2 weeks before. Prior radiotherapy to major organs within 2 weeks of admission, or focal radiotherapy, such as to bones, limbs, or other areas not involving major organs, within 3 days.
- Patients who have clinically significant, uncontrolled, cardiovascular disease, including congestive heart failure Grades II, III or IV according to the New York Heart Association classification, myocardial infarction or unstable angina within the previous 6 months, or uncontrolled hypertension.
- Patients who have experienced arterial thrombotic or embolic events such as cerebrovascular accident (including transient ischemic attacks), or pulmonary embolism within the 6 months before randomization or venous thrombotic events such as deep vein thrombosis within the 3 months before screening.
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- Patients who have experienced any hemorrhage or bleeding event NCI CTCAE version 5.0 Grade 3 or higher within 4 weeks before screening.
- Patients who have a known risk of intracranial bleeding, such as a brain aneurysm or history of intracranial bleeding within 1 year prior to screening.
- Patients who have a non-healing wound, ulcer, or bone fracture.
- Patients who have poor organ function as defined by one or more of the following laboratory parameters:
- Persistent proteinuria of NCI-CTCAE version 4.03 Grade 3 or higher
- Alanine aminotransferase and aspartate aminotransferase (AST) > 3 × upper limit of normal (ULN) if no hepatic metastases are present; > 5 × ULN if hepatic metastases are present.
- Total bilirubin >1.5 × ULN; and in presence of Gilbert's syndrome, total bilirubin > 3 × ULN or direct bilirubin > 1.5 × ULN.
- Estimated (Cockcroft-Gault formula) or measured creatinine clearance < 40 mL/min.
- Platelet count < 90 × 109/L and absolute neutrophil count (ANC) < 1.0 × 10^9/L.
- Hemoglobin < 9 g/dL. Transfusion and erythropoietin may be used to reach at least 9 g/dL, but must have been administered at least 2 weeks before screening.
- Patients who have received neutrophil growth factor support within 14 days of screening.
- Patients who require therapy with a concomitant medication that is a strong inhibitor or strong inducer of CYP3A4.
- Patients who have had a major surgical procedure (minor surgical procedures such as central venous catheter placement, tumor needle biopsy, and feeding tube placement are not considered major surgical procedures) within 14 days of screening. Patient has significant traumatic injury within 28 days before screening.
- Patients who have a history of another primary malignancy that has been diagnosed or required therapy within 2 years before screening. (The following are exempt from the 2-year limit: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site.)
- Patients who have a history of a seizure disorder requiring anti-seizure medication.
- Patients who have metastases to the brain.
- Patients who are unwilling or unable to comply with scheduled visits, drug administration plan, laboratory tests, or other study procedures and study restrictions.
- Patients who have a QT interval corrected using Fridericia's formula (QTcF) of > 450 msec.
- Women who are unwilling, if not postmenopausal or surgically sterile, to abstain from sexual intercourse or employ highly effective contraception from the time of randomization and for at least 30 days after the last dose of study drug. Men who are unwilling, if not surgically sterile, to abstain from sexual intercourse or employ highly effective contraception from the time of randomization and for at least 90 days after the last dose of study drug. Refer to Appendix G for acceptable methods of contraception.
- Women who are pregnant, as documented by a serum beta human chorionic gonadotropin (β-hCG) pregnancy test consistent with pregnancy, obtained within 7 days before the treatment assignment. Females with β-hCG values that are within the range for pregnancy but are not pregnant (false positives) may be enrolled with written consent of the investigator, after pregnancy has been ruled out. Females of non-childbearing potential (postmenopausal for more than 1 year; bilateral tubal ligation; bilateral oophorectomy; hysterectomy) do not require a serum β-hCG test.
- Women who are breastfeeding.
- Patients who have prior or ongoing clinically significant illness, medical condition, surgical history, physical finding, or laboratory abnormality that, in the Investigator's opinion, could affect the safety of the patient; alter the absorption, distribution, metabolism, or excretion of the study drug; or impair the assessment of study results.
- Patients with impaired decision-making capacity.
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
University of Miami
Miami, Florida, 33136
Leonela Wright, 305-243-0864, lxw612@med.miami.edu
Jonathan Trent, MD, PhD, 305-243-2581, JTrent@med.miami.edu
About this study
Official title: ctDNA-Guided Sunitinib And Regorafenib Therapy for Gastrointestinal Stromal Tumor (GIST)
The purpose of this research is to test if mutations (changes in DNA) in exons (segment of DNA or RNA containing information that has the instructions for making proteins) in the KIT gene can be used to predict the body's response to standard of care treatment.
- Conditions
- Gastrointestinal Stromal Tumors
- Being tested
- Sunitinib, Regorafenib
- Medical subject headings
- Gastrointestinal Stromal Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SunitinibDrug
Also called Sutent, SU11248
37.5 mg of Sunitinib orally (PO), once daily on a 28-day treatment cycle.
RegorafenibDrug
Also called Stivarga, Regonix, BAY 73-4506
120.0 mg of Regorafenib, once daily by mouth 3 weeks on 1 week off on a 4-week treatment cycle.
| Study group | Role | What they receive |
|---|---|---|
| Group A: KIT Exon 13 receiving Sunitinib | Experimental | Participants with KIT mutation on exon 13 will receive Sunitinib. Participants showing disease progression after first-assigned Sunitinib therapy have the option to receive Regorafenib therapy. Total allotted time for treatment is up to 12 months. Receives: Drug: Sunitinib; Drug: Regorafenib |
| Group B: KIT Exon 17 receiving Regorafenib | Experimental | Participants with KIT mutation on exon 17 will receive Regorafenib. Participants showing disease progression after first-assigned Regorafenib therapy have the option to receive Sunitinib therapy. Total allotted time for treatment is up to 12 months. Receives: Drug: Sunitinib; Drug: Regorafenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Achieving Overall Response
The overall response rate (ORR) will be defined as the number of evaluable patients whose best overall response to study treatment is complete response (CR) or partial response (PR). ORR will be assessed using Choi criteria by treating physician.
Measured: Up to 12 months
Secondary outcomes (4)
Progression-Free Survival (PFS)
Progression-free survival (PFS) will be defined as the time elapsed from the start of treatment to the date of documented progression or death, whichever comes first. For surviving patients without progression who begin alternative treatment, PFS will be censored at the last date of documented progression-free status prior to starting alternative treatment. Similarly, losses to follow up will be censored at the last date of documented progression-free status.
Measured: Up to 3 years
Overall Survival (OS)
Overall survival (OS) will be defined as the time elapsed from the start of treatment until death. For surviving patients, follow-up will be censored at the date of last contact.
Measured: Up to 3 years
Number of Participants Achieving Clinical Benefit
The clinical benefit rate (CBR) will be defined as the number of evaluable patients whose best overall response to study treatment is complete response (CR), or partial response (PR), or stable disease (SD), for 6 months or more and will be assessed using Choi criteria by treating physician.
Measured: Up to 12 months
Incidence of Treatment-Related Toxicity and Adverse Events
Safety and tolerability of assigned Sunitinib and Regorafenib treatment will be reported as the incidence of treatment-related toxicity, including serious adverse events (SAEs) and adverse events (AEs), in study participants using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0, per physician discretion.
Measured: Up to 13 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 May 2022
- Study start17 Oct 2023
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gastrointestinal Stromal TumorsStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Miami
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jonathan Trent, MD, PhD, University of Miami
- Investigators
- Jonathan Trent, MD, PhD, Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05366816
- Sponsor's protocol ID
- 20201368
- Status last verified
- Oct 2026
- First submitted
- 5 May 2022
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ctDNA-Guided Sunitinib And Regorafenib Therapy for Gastrointestinal Stromal Tumor (GIST)
- ClinicalTrials.gov ID
- NCT05366816
- Acronym / sponsor ID
- 20201368
- Conditions
- Gastrointestinal Stromal Tumors
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- University of MiamiUniversity, hospital or other
- Study start
- 17 Oct 2023 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 9 May 2022 (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.
The purpose of this research is to test if mutations (changes in DNA) in exons (segment of DNA or RNA containing information that has the instructions for making proteins) in the KIT gene can be used to predict the body's response to standard of care treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A: KIT Exon 13 receiving Sunitinib | Experimental | Drug: Sunitinib, Drug: RegorafenibParticipants with KIT mutation on exon 13 will receive Sunitinib. Participants showing disease progression after first-assigned Sunitinib therapy have the option to receive Regorafenib therapy. Total allotted time for treatment is up to 12 months. |
| Group B: KIT Exon 17 receiving Regorafenib | Experimental | Drug: Sunitinib, Drug: RegorafenibParticipants with KIT mutation on exon 17 will receive Regorafenib. Participants showing disease progression after first-assigned Regorafenib therapy have the option to receive Sunitinib therapy. Total allotted time for treatment is up to 12 months. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients who are ≥ 18 years of age.
- Patients who have histologically confirmed metastatic or unresectable GIST. Unresectable GIST must be confirmed to be unresectable by an experienced surgeon.
- Patients may have had any prior lines of TKI therapy.
- Patients who received imatinib prior treatment regimens, including adjuvant therapy, with objective disease progression, inadequate clinical benefit, or intolerance. Additionally, disease progression or intolerance to other systemic therapies including investigational agents and radiotherapy is allowed. --- Patients who experienced intolerance to prior therapies must have objective disease progression prior to enrollment.
- Patients who have an ECOG PS of 0 to 2.
Key exclusion criteria (29 listed)
- Patients who have received prior treatment with sunitinib or regorafenib.
- Patients who have received more than 2 different prior tyrosine kinase inhibitor (TKI) treatment regimens. If a patient receives the same TKI more than once sequentially (eg, imatinib followed by a period without systemic therapy and retreatment with imatinib), that will be counted as a single TKI treatment regimen.
- Patients who are known to be KIT wild type.
- Patients who received any systemic anticancer therapy within 2 weeks before. Prior radiotherapy to major organs within 2 weeks of admission, or focal radiotherapy, such as to bones, limbs, or other areas not involving major organs, within 3 days.
- Patients who have clinically significant, uncontrolled, cardiovascular disease, including congestive heart failure Grades II, III or IV according to the New York Heart Association classification, myocardial infarction or unstable angina within the previous 6 months, or uncontrolled hypertension.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SunitinibAlso: Sutent, SU1124837.5 mg of Sunitinib orally (PO), once daily on a 28-day treatment cycle. | Drug | Group A: KIT Exon 13 receiving Sunitinib, Group B: KIT Exon 17 receiving Regorafenib |
| RegorafenibAlso: Stivarga, Regonix, BAY 73-4506120.0 mg of Regorafenib, once daily by mouth 3 weeks on 1 week off on a 4-week treatment cycle. | Drug | Group A: KIT Exon 13 receiving Sunitinib, Group B: KIT Exon 17 receiving Regorafenib |
Primary outcome
Number of Participants Achieving Overall Response
Time frame: Up to 12 months
The overall response rate (ORR) will be defined as the number of evaluable patients whose best overall response to study treatment is complete response (CR) or partial response (PR). ORR will be assessed using Choi criteria by treating physician.
4 secondary outcomes
Progression-Free Survival (PFS)
Time frame: Up to 3 years
Overall Survival (OS)
Time frame: Up to 3 years
Number of Participants Achieving Clinical Benefit
Time frame: Up to 12 months
Incidence of Treatment-Related Toxicity and Adverse Events
Time frame: Up to 13 months
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 23 Oct 2026.
- Individual participant dataSharing planned: No
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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.
Jonathan Trent, MD, PhD principal investigator
University of Miami
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sunitinib and Regorafenib in people with Gastrointestinal Stromal Tumors, run by University of Miami 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 7 inclusion and 29 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 23 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Leonela Wright (305-243-0864, lxw612@med.miami.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Achieving Overall Response (time frame: Up to 12 months). 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 23 Oct 2026.