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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 de abril de 2026 actualizado por: 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.

Descripción general del estudio

Descripción detallada

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 de estudio

Intervencionista

Inscripción (Estimado)

100

Fase

  • Fase 2

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

  • Nombre: Jian Li, MD
  • Número de teléfono: +86 010 88196561
  • Correo electrónico: oncogene@163.com

Ubicaciones de estudio

    • Beijing Municipality
      • Beijing, Beijing Municipality, Porcelana, 100101
        • Reclutamiento
        • Beijing Cancer Hospital
        • Investigador principal:
          • Jian Li, MD
        • Contacto:
          • Jian Li, MD
          • Número de teléfono: +86 010-88196561
          • Correo electrónico: oncogene@163.com

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

  • Adulto
  • Adulto Mayor

Acepta Voluntarios Saludables

No

Descripción

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.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: Aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Comparador activo: 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
Experimental: 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.

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Progression Free Survival(PFS)
Periodo de tiempo: 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

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Investigadores

  • Silla de estudio: Jian Li, MD, Peking University Cancer Hospital & Institute

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

14 de julio de 2025

Finalización primaria (Estimado)

1 de junio de 2027

Finalización del estudio (Estimado)

1 de junio de 2027

Fechas de registro del estudio

Enviado por primera vez

20 de abril de 2026

Primero enviado que cumplió con los criterios de control de calidad

27 de abril de 2026

Publicado por primera vez (Actual)

30 de abril de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

30 de abril de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

27 de abril de 2026

Última verificación

1 de abril de 2026

Más información

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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