Questa pagina è stata tradotta automaticamente e l'accuratezza della traduzione non è garantita. Si prega di fare riferimento al Versione inglese per un testo di partenza.

Comparing Regorafenib Combined With Envafolimab to Physician's Choice in Patients With Metastatic Gastrointestinal Stromal Tumors Harboring KIT Exon 17 Mutations Refractory to Standard Treatment

27 aprile 2026 aggiornato da: Jian Li

A Phase II, Open-label, Multicenter, Randomized Controlled Clinical Trial Comparing Regorafenib Combined With Envafolimab to Physician's Choice in Patients With Metastatic Gastrointestinal Stromal Tumors Harboring KIT Exon 17 Mutations Refractory to Standard Treatment

Gastrointestinal stromal tumors (GISTs) are mostly driven by c-kit or PDGFRA mutations, and commonly occur in the stomach and small intestine. Targeted therapy is the mainstay for advanced metastatic GISTs, but there is a lack of effective regimens after drug resistance. Immune checkpoint inhibitors (ICIs) have limited efficacy as monotherapy, while tyrosine kinase inhibitor (TKI) drugs such as regorafenib can improve the immune microenvironment and exert a synergistic effect when combined with ICIs, with more significant efficacy especially in cases with kit exon 17 mutations. This study aims to explore the effectiveness of regorafenib combined with envafolimab in metastatic gastrointestinal stromal tumors with kit exon 17 mutations that have failed standard treatment.

Panoramica dello studio

Stato

Reclutamento

Condizioni

Intervento / Trattamento

Descrizione dettagliata

This is a multicenter, prospective phase II clinical study aiming to explore the efficacy and safety of regorafenib combined with envafolimab in metastatic gastrointestinal stromal tumors (GIST) with KIT exon 17 mutations that have failed standard treatment, as well as to investigate the correlation between the immune microenvironment and the efficacy of immunotherapy.

The study will enroll patients with histologically confirmed advanced metastatic GIST harboring KIT exon 17 mutations, with at least one evaluable lesion. Patients will be randomized at a 1:1 ratio into two groups: the experimental group will receive regorafenib combined with envafolimab (regorafenib at a recommended dose of 120 mg orally once daily, administered for 3 weeks followed by 1 week off; envafolimab 200 mg subcutaneously injected once every 2 weeks); the control group will receive either re-challenge with one previously effective targeted agent or combination therapy with two agents selected based on the clinician's experience. Treatment will continue until disease progression, occurrence of intolerable toxicity, or voluntary withdrawal of the patient from the trial.

A total of 100 patients are planned to be enrolled. Enrolled patients will undergo imaging assessments at baseline and every 2 months during treatment.

Tipo di studio

Interventistico

Iscrizione (Stimato)

100

Fase

  • Fase 2

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Contatto studio

  • Nome: Jian Li, MD
  • Numero di telefono: +86 010 88196561
  • Email: oncogene@163.com

Luoghi di studio

    • Beijing Municipality
      • Beijing, Beijing Municipality, Cina, 100101
        • Reclutamento
        • Beijing Cancer Hospital
        • Investigatore principale:
          • Jian Li, MD
        • Contatto:

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Adulto
  • Adulto più anziano

Accetta volontari sani

No

Descrizione

Inclusion Criteria:

  • Aged ≥ 18 years, regardless of gender;
  • Confirmed as gastrointestinal stromal tumor by histopathological examination;
  • Having at least one measurable target lesion meeting the criteria of mRECIST v1.1 (non-lymph node lesions with a long axis ≥ 1.0 cm or a long axis ≥ the thickness of 2 slides); imaging assessment should be performed within 14 days before the first medication;
  • Disease progression or intolerance after treatment with imatinib, sunitinib, regorafenib, or ripretinib;
  • Primary or secondary KIT exon 17 mutation detected by genetic testing;
  • Sufficient organ and bone marrow function, defined as follows: Blood routine: Absolute neutrophil count (ANC) ≥ 1.5×10⁹/L; platelet count (PLT) ≥ 75×10⁹/L; hemoglobin (HGB) ≥ 9.0 g/dL. No use of granulocyte colony-stimulating factor (G-CSF), granulocyte-macrophage colony-stimulating factor (GM-CSF), red blood cell transfusion, or platelet transfusion within 14 days before the examination; Liver and kidney function: For subjects without liver metastasis, serum total bilirubin (TBIL) ≤ 1.5×upper limit of normal (ULN); alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5×ULN. For subjects with liver metastasis: TBIL ≤ 1.5×ULN; ALT and AST ≤ 5×ULN. Renal function: Serum creatinine (Scr) ≤ 1.5×ULN; Sufficient coagulation function, defined as international normalized ratio (INR) ≤ 1.5 or prothrombin time (PT) ≤ 1.5×ULN; if the subject is receiving anticoagulant therapy, it is acceptable as long as PT is within the range specified for the anticoagulant drug;
  • Provide 15 paraffin-embedded tissue sections before enrollment for immune microenvironment detection;
  • ECOG PS score of 0-2;
  • Signed informed consent form.

Exclusion Criteria:

  • A history of intolerance to regorafenib treatment, or previous receipt of immune checkpoint inhibitor therapy;
  • Being in pregnancy or lactation;
  • Those with an expected survival period of less than 3 months;
  • Those who have undergone major surgery or suffered significant trauma within 4 weeks before the first blood collection during the screening period, or are expected to require major surgery during the study period;
  • Patients with current active ulcers or gastrointestinal bleeding;
  • A history of interstitial lung disease or non-infectious pneumonia; a history of active tuberculosis;
  • Uncontrolled pleural effusion, pericardial effusion, or ascites requiring repeated drainage;
  • Patients with clinically confirmed autoimmune diseases; those with positive HIV or HCV; those with HBV-DNA exceeding the laboratory normal range; those with acute CMV infection;
  • Patients with clinically confirmed central nervous system metastases;
  • Patients with other malignant tumors within 5 years;
  • Immunosuppressed subjects, including those with known immunodeficiency; those currently receiving systemic steroid medications (except those who have used inhaled steroids recently or currently);
  • Uncontrolled hypertension: After active antihypertensive treatment, three consecutive blood pressure measurements indicate systolic blood pressure ≥ 160 mmHg and diastolic blood pressure ≥ 100 mmHg;
  • Subjects who, in the investigator's assessment, are unable or unwilling to comply with the requirements of the study protocol.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Randomizzato
  • Modello interventistico: Assegnazione parallela
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Comparatore attivo: control group(physician-selected)
Physicians will make selections based on previous drug tolerance, genetic types, etc.: 1) Maintenance treatment with the original dose of previously effective TKIs: during the previous treatment, the patient had achieved at least stable disease (SD) or partial response (PR), with progression-free survival (PFS) exceeding 6 months and tolerable adverse reactions; 2) Combination therapy with two TKI targeted drugs: according to different action targets indicated by genetic testing of the patient's tissue or peripheral blood, different drugs will be selected for maintenance, or a combination of previously effective and well-tolerated drugs will be chosen, or combination therapy will be selected with reference to previous tolerance.
physician-selected treatment
Sperimentale: treatment group(Regorafenib + Envafolimab)
Patients in the treatment group will receive regorafenib combined with envafolimab. Regorafenib: 120 mg, orally, once daily, administered for 3 weeks followed by 1 week of rest. Envafolimab: 200 mg, subcutaneously injected, once every 2 weeks.

Regorafenib: 120mg, orally, qd, administered for 3 weeks followed by 1 week of rest.

Envafolimab: 200mg, subcutaneously injected, q21d.

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Progression Free Survival(PFS)
Lasso di tempo: Up to 2 years
Defined as the time from date of study treatment to disease progression radiological/clinical or death due to any cause, whichever occurs first.
Up to 2 years

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Investigatori

  • Cattedra di studio: Jian Li, MD, Peking University Cancer Hospital & Institute

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

14 luglio 2025

Completamento primario (Stimato)

1 giugno 2027

Completamento dello studio (Stimato)

1 giugno 2027

Date di iscrizione allo studio

Primo inviato

20 aprile 2026

Primo inviato che soddisfa i criteri di controllo qualità

27 aprile 2026

Primo Inserito (Effettivo)

30 aprile 2026

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

30 aprile 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

27 aprile 2026

Ultimo verificato

1 aprile 2026

Maggiori informazioni

Termini relativi a questo studio

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

No

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

prodotto fabbricato ed esportato dagli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .