Evaluating drug holidays for high-risk GIST patients based on MRD detection

Clinical Research of Drug Holiday Based on MRD Detection in Gastrointestinal Stromal Tumor Patients at High Risk of Recurrence

This study is testing if high-risk patients with gastrointestinal stromal tumors can safely take breaks from their imatinib treatment based on blood tests that check for leftover cancer cells.

Quick facts

• Study type: Observational
• Enrollment: 40 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Peking University People's Hospital (Academic / other)
• Drugs / interventions: imatinib
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT05867901 on ClinicalTrials.gov

What this trial studies

This observational study focuses on patients with gastrointestinal stromal tumors (GIST) who are at high risk of recurrence after surgery. It aims to assess the value of monitoring minimal residual disease (MRD) through circulating tumor DNA (ctDNA) to determine the timing of drug withdrawal from imatinib, a common treatment. The study will evaluate the effects of a 'drug holiday' on progression-free survival and overall survival, as well as the potential for reusing imatinib after disease progression. The goal is to identify which patients can safely pause treatment without compromising their health outcomes.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-75 who have undergone surgery for high-risk GIST and have been on imatinib for at least three years with no detectable disease.

Not a fit: Patients with primary imatinib resistant mutations or those who have not achieved disease control after surgery may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could allow patients to safely take breaks from medication while maintaining disease control.

How similar studies have performed: Previous studies have shown promise in using MRD detection for treatment decisions in other cancers, suggesting potential for success in this novel approach for GIST.

Eligibility criteria

Inclusion Criteria:

* ① Age: ≥18 years and ≤75 years;
* ② The primary lesion (and metastatic lesion) underwent R0 or R1 surgical resection, and patients were clinically diagnosed with high recurrence risk GIST combined with postoperative pathological results;
* ③ The patient have received imatinib adjuvant therapy for 3 years after surgery, and no gross lesions were detected by imaging examination, and the possibility of drug withdrawal was considered based on the comprehensive judgment of the doctor in charge;
* ④ The liquid biopsy for baseline ctDNA is negative;
* ⑤ Primary Imatinib resistant mutations were not detected by genetic testing, such as SDH-deficient GIST, NF1-mutant GIST, BRAF-mutant GIST and GIST with NTRK3 rearrangement;
* ⑥ PS score is 0-1 and expected survival time is more than 4 months;
* ⑦ Patients are willing to stop drug use and observe, and patients and their families could understand the study protocol and voluntarily participate in this study, and signed informed consent. In the follow-up study, they could provide the clinical pathological data and imaging data needed for the study process, cooperate with the follow-up and collect the blood of the clinical efficacy evaluation, and agree to use the test data for the follow-up study and product development.

Exclusion Criteria:

* ① Fresh or paraffin tissue of the surgical tumor is not available;
* ② Not receiving the assigned treatment or change therapy before disease progression;
* ③ Unable to cooperate with follow-up with the study according to defined clinical period;
* ④ Unable to take or provide defined imaging assessment examinations;
* ⑤ Other conditions that investigators consider inappropriate for enrollment.

Where this trial is running

Beijing, Beijing Municipality

• Peking University People's Hospital — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Principal investigator: Zhidong Gao, MD — Peking University People's Hospital
• Study coordinator: Zhidong Gao, MD

Conditions Gastrointestinal Stromal Tumors; Drug Holiday; Minimal Residual Disease; Circulating Tumor DNA