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An Umbrella-designed Prospective Multicenter Randomized Open-label Superiority Trial Evaluating Multi-biomarker-guided Precision Therapy for First-line Treatment of Advanced Esophageal Squamous Cell Carcinoma

13 de julio de 2026 actualizado por: Rui-hua Xu, MD, PhD, Sun Yat-sen University

A Prospective, Multicenter, Randomized, Open-label, Superiority Clinical Trial With an Umbrella Trial Design Framework to Evaluate the Efficacy of a Precision Treatment Strategy Guided by Multiple Biomarkers in the First-line Therapy of Advanced Esophageal Squamous Cell Carcinoma

This is a prospective, multicenter, randomized, open-label, umbrella superiority clinical trial for patients with advanced metastatic esophageal squamous cell carcinoma who have not received prior anti-tumor treatment.

All eligible participants will be randomly assigned into two cohorts: Cohort A (standard treatment group) and Cohort B (biomarker-guided precision treatment group).

Patients in Cohort A will receive first-line standard therapy consisting of TP chemotherapy plus PD-1 inhibitor.

All patients in Cohort B will first complete three biomarker tests, then be divided into 4 sub-groups based on biomarker results to receive biomarker-directed additional treatment combined with the same standard backbone therapy. Participants with all negative biomarkers or failed biomarker testing will receive the identical standard treatment as Cohort A.

The primary objective is to compare the survival benefit between biomarker-guided multi-strategy precision therapy and conventional standard first-line treatment.

Descripción general del estudio

Descripción detallada

This umbrella-design randomized controlled trial enrolls treatment-naïve patients aged 18-75 years with histologically confirmed metastatic esophageal squamous cell carcinoma with measurable lesions per RECIST 1.1 and ECOG PS 0-1.

Subjects are randomized at a 1:3.75 ratio to standard first-line TP+PD-1 treatment (Cohort A) or biomarker-stratified experimental treatment (Cohort B). Three predictive biomarkers including plasma TGF-β, circulating metabolite score and blood Lactobacillus level will be detected at screening for Cohort B patients to allocate targeted add-on interventions.

Key exclusion criteria include uncontrolled brain metastasis, severe organ dysfunction, active severe infection, prior immune checkpointInhibitor treatment and bleeding disorders with a high risk of hemorrhage. The main efficacy endpoint is progression-free survival; secondary endpoints include objective response rate, disease control rate, overall survival, safety and so on.

Tipo de estudio

Intervencionista

Inscripción (Estimado)

347

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: XU ruihua Xuruihua
  • Número de teléfono: 020-87342479
  • Correo electrónico: luohy@sysucc.org.cn

Ubicaciones de estudio

    • GUANGZHOU
      • Guangdong, GUANGZHOU, Porcelana, 510000
        • Sub-i
        • Contacto:
          • Luo huiyan Luohuiyan
          • Número de teléfono: 02087343560
          • Correo electrónico: luohy@sysucc.org.cn

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:

  1. Aged between 18 and 75 years (calculated on the date of signing the informed consent form);
  2. Patients with metastatic esophageal squamous cell carcinoma (ESCC) confirmed by histopathology or cytology;
  3. Treatment-naïve patients with no prior anti-tumor therapy;
  4. Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 or 1, with an expected survival of more than 3 months;
  5. Judged by the investigator to be suitable for first-line chemotherapy;
  6. Presence of at least one measurable lesion as defined by the Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1);
  7. Women of childbearing potential must agree to use effective contraception (e.g., intrauterine device, oral contraceptives, condoms) throughout the study period and for 6 months after the end of study treatment; they must have a negative serum or urine pregnancy test within 7 days prior to enrollment and shall not be breastfeeding. Male patients must agree to use effective contraception throughout the study period and for 6 months after the end of study treatment.

    Version: 1.0, Version Date: March 18, 2026

  8. Voluntarily participate in this study and provide written informed consent.

Exclusion Criteria:

  1. Brain metastases with symptoms or symptom control duration less than 2 months;
  2. History of or concurrent other malignant tumors within the past 3 years (excluding cured basal cell carcinoma of the skin and carcinoma in situ of the cervix);
  3. Insufficient bone marrow hematopoietic function (without blood transfusion within 14 days):

    1. Absolute Neutrophil Count (ANC) < 1.5 × 10⁹/L;
    2. Platelet count < 100 × 10⁹/L;
    3. Hemoglobin < 90 g/L.
  4. Hepatic abnormalities:

    1. Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST) or Alkaline Phosphatase (ALP) > 2.5 × Upper Limit of Normal (ULN) in patients without liver metastases; ALT, AST or ALP > 5 × ULN in patients with liver metastases;
    2. Serum total bilirubin > 1.5 × ULN (> 3 × ULN for patients with Gilbert's syndrome);
    3. Decompensated liver cirrhosis (Child-Pugh liver function grade B or C);
    4. Positive Hepatitis B Surface Antigen (HBsAg) with Hepatitis B Virus (HBV) DNA load ≥ 2000 IU/mL. Patients with positive HBsAg and HBV DNA load < 2000 IU/mL must receive anti-HBV therapy for at least 2 weeks prior to the first study drug administration;
    5. Positive Hepatitis C Virus (HCV) antibody with detectable HCV RNA.
  5. Renal abnormalities:

    1. Serum creatinine > 1.5 × ULN or estimated creatinine clearance < 60 mL/min calculated by the Cockcroft-Gault formula;
    2. Urinalysis showing urine protein ≥ ++, confirmed with 24-hour urinary protein quantification > 1.0 g;
    3. Renal failure requiring hemodialysis or peritoneal dialysis;
    4. Medical history of nephrotic syndrome.
  6. Bleeding risks:

    1. Abnormal coagulation function: Activated Partial Thromboplastin Time (APTT) or Thrombin Time (TT) > 1.5 × ULN, or International Normalized Ratio (INR) > 1.5 (> 2.5 for subjects receiving anticoagulant therapy);
    2. History of hemorrhage (hemoptysis), coagulopathy, or ongoing use of warfarin, aspirin, low-molecular-weight heparin and other antiplatelet drugs (except prophylactic aspirin at a dose ≤ 100 mg/day);
    3. Any signs or history of bleeding diathesis regardless of severity;
    4. Active gastrointestinal bleeding defined as hematemesis, hematochezia or melena within the past 3 months without evidence of resolution confirmed by gastroscopy or colonoscopy;
    5. Any Grade ≥ 3 bleeding event per CTCAE occurring within 4 weeks prior to the first study drug administration.

    Version: 1.0, Version Date: March 18, 2026

  7. Cardiovascular and cerebrovascular abnormalities:

    1. Subjects with any of the following conditions within 12 months before the first study drug administration: grade ≥ II myocardial ischemia or myocardial infarction, arrhythmia, grade ≥ III cardiac insufficiency, uncontrolled angina, coronary/peripheral artery bypass graft surgery, symptomatic congestive heart failure, cerebrovascular accident or transient ischemic attack, etc.;
    2. Deep vein thrombosis or pulmonary embolism occurring within 6 months before the first study drug administration;
    3. Left Ventricular Ejection Fraction (LVEF) < 50% assessed by Doppler echocardiography;
    4. Average Fridericia-corrected QT interval (QTcF) (from at least 3 consecutive electrocardiograms): ≥ 450 ms for male patients, ≥ 470 ms for female patients;
    5. Uncontrolled hypertension refractory to medication (systolic blood pressure ≥ 150 mmHg and diastolic blood pressure ≥ 100 mmHg recorded in at least two measurements).
  8. History of immunodeficiency:

    1. Confirmed Human Immunodeficiency Virus (HIV) infection;
    2. Other acquired or congenital immunodeficiency disorders;
    3. Scheduled or prior solid organ transplantation, hematopoietic stem cell transplantation within 60 days before the first study drug administration, or significant graft-versus-host disease;
    4. Patients requiring immunosuppressants, systemic or absorbable local hormonal therapy for immunosuppressive purposes that must be continued within 7 days before the first study drug administration (excluding daily glucocorticoid dose < 10 mg prednisone or equivalent steroids).
  9. Active or uncontrolled severe infection (Grade ≥ 2 per CTCAE);
  10. Uncontrolled pleural effusion, pericardial effusion or ascites requiring repeated drainage (judged by the investigator);
  11. Prior treatment with any anti-PD-1, anti-PD-L1, anti-PD-L2 or anti-CTLA-4 antibody, or any other antibodies targeting T cell co-stimulatory or checkpoint pathways (e.g., OX40, CD137, etc.);
  12. Complicated with severe or poorly controlled diseases judged by the investigator to carry substantial risks for study participation (e.g., poorly controlled diabetes with screening fasting plasma glucose (FPG) > 10 mmol/L);
  13. Participation in another clinical trial of anti-tumor agents within 28 days prior to screening.

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: Conventional Therapy

Participants in this arm will receive standard first-line treatment for advanced esophageal squamous cell carcinoma, consisting of TP chemotherapy plus a PD-1 inhibitor.

TP regimen refers to Nab-Paclitaxel combined with cisplatin.

Paclitaxel + Cisplatin (TP regimen) plus PD-1 inhibitor
Experimental: Experimental Arm

Subgroup B1 (TGF-β-guided Intervention):

Patients with positive plasma TGF-β levels detected by ELISA; they will receive Retlirafusp alfa combined with chemo-immunotherapy backbone.

Subgroup B2 (Metabolite-guided Intervention):

Patients who do not meet the criteria for Subgroup B1 and have positive plasma metabolite predictive score; they will receive indole-3-carbinol plus fermented black garlic extract combined with chemo-immunotherapy backbone.

Subgroup B3 (Lactobacillus-guided Intervention):

Patients who do not meet the criteria for Subgroup B1 or B2 and have positive blood Lactobacillus levels measured by qPCR; they will receive Lactobacillus rhamnosus combined with chemo-immunotherapy backbone.

Subgroup B4 (Screening Failure Subgroup):

Patients with negative results for all three biomarkers due to technical issues; they will receive the same conventional treatment as Cohort A.

Group B1 (TGF-guided intervention, n=73): Plasma TGF-B ELISA levels positive, combined with Retlirafusp alfa on top of chemo-immunotherapy backbone; Group B2 (metabolite-guided intervention, n=73): Did not meet TGF-β positivity, combined with indole-3-carbinol and black garlic fermentation extract on top of chemo-immunotherapy backbone; Group B3 (Lactobacillus-guided intervention, n=73): Those who don't meet B1 or B2 criteria but have positive blood Lactobacillus qPCR levels, combined with Lactobacillus rhamnosus on top of chemo-immunotherapy backbone; Group B4 (Screening failed group, n=55): All three biomarker tests are negative or testing failed due to technical reasons, receive the same conventional treatment as cohort A

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Periodo de tiempo
Progression-Free Survival (PFS)
Periodo de tiempo: Up to 2 Years
Up to 2 Years

Medidas de resultado secundarias

Medida de resultado
Periodo de tiempo
Duración de la respuesta (DOR)
Periodo de tiempo: hasta 2 años
hasta 2 años
Tasa de Control de Enfermedades (DCR)
Periodo de tiempo: hasta 2 años
hasta 2 años
Supervivencia general (SG)
Periodo de tiempo: hasta 2 años
hasta 2 años
Objective Response Rate (ORR)
Periodo de tiempo: Up to 2 Years
Up to 2 Years
quality of life score
Periodo de tiempo: up to 2 years
up to 2 years
incidence and severity of adverse events (AEs)
Periodo de tiempo: up to 2 years
up to 2 years
serious adverse events (SAEs)
Periodo de tiempo: up to 2 years
up to 2 years

Otras medidas de resultado

Medida de resultado
Medida Descripción
Periodo de tiempo
Correlation analysis between TGFβ and Incidence of treatment response
Periodo de tiempo: up to 2 years
TGFβ concentration is detected by plasma ELISA
up to 2 years
Correlation analysis between Metabolite Score and Incidence of treatment response
Periodo de tiempo: up to 2 years
Metabolite Score is detected by plasma metabolome
up to 2 years
Correlation analysis between blood Lactobacillus qPCR levels and Incidence of treatment response
Periodo de tiempo: up to 2 years
up to 2 years

Colaboradores e Investigadores

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

Patrocinador

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 (Estimado)

7 de julio de 2026

Finalización primaria (Estimado)

30 de junio de 2029

Finalización del estudio (Estimado)

30 de junio de 2029

Fechas de registro del estudio

Enviado por primera vez

6 de julio de 2026

Primero enviado que cumplió con los criterios de control de calidad

13 de julio de 2026

Publicado por primera vez (Actual)

16 de julio de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

16 de julio de 2026

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

13 de julio de 2026

Última verificación

1 de julio de 2026

Más información

Términos relacionados con este estudio

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

SÍ

Descripción del plan IPD

Study ProtocolStatistical Analysis Plan (SAP)Informed Consent Form (ICF)

Marco de tiempo para compartir IPD

The data can be shared when the study is finished and ready for publication.

Criterios de acceso compartido de IPD

PI

Tipo de información de apoyo para compartir IPD

  • PROTOCOLO DE ESTUDIO
  • SAVIA
  • CIF

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

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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