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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 juillet 2026 mis à jour par: 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.

Aperçu de l'étude

Description détaillée

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.

Type d'étude

Interventionnel

Inscription (Estimé)

347

Phase

  • Phase 2

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Coordonnées de l'étude

Lieux d'étude

    • GUANGZHOU
      • Guangdong, GUANGZHOU, Chine, 510000
        • Sub-i
        • Contact:

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

  • Adulte
  • Adulte plus âgé

Accepte les volontaires sains

Non

La description

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 d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Randomisé
  • Modèle interventionnel: Affectation parallèle
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Comparateur actif: 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
Expérimental: 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

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Délai
Progression-Free Survival (PFS)
Délai: Up to 2 Years
Up to 2 Years

Mesures de résultats secondaires

Mesure des résultats
Délai
Durée de la réponse (DOR)
Délai: jusqu'à 2 ans
jusqu'à 2 ans
Taux de contrôle de la maladie (DCR)
Délai: jusqu'à 2 ans
jusqu'à 2 ans
Survie globale (SG)
Délai: jusqu'à 2 ans
jusqu'à 2 ans
Objective Response Rate (ORR)
Délai: Up to 2 Years
Up to 2 Years
quality of life score
Délai: up to 2 years
up to 2 years
incidence and severity of adverse events (AEs)
Délai: up to 2 years
up to 2 years
serious adverse events (SAEs)
Délai: up to 2 years
up to 2 years

Autres mesures de résultats

Mesure des résultats
Description de la mesure
Délai
Correlation analysis between TGFβ and Incidence of treatment response
Délai: 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
Délai: 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
Délai: up to 2 years
up to 2 years

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Estimé)

7 juillet 2026

Achèvement primaire (Estimé)

30 juin 2029

Achèvement de l'étude (Estimé)

30 juin 2029

Dates d'inscription aux études

Première soumission

6 juillet 2026

Première soumission répondant aux critères de contrôle qualité

13 juillet 2026

Première publication (Réel)

16 juillet 2026

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

16 juillet 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

13 juillet 2026

Dernière vérification

1 juillet 2026

Plus d'information

Termes liés à cette étude

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

OUI

Description du régime IPD

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

Délai de partage IPD

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

Critères d'accès au partage IPD

PI

Type d'informations de prise en charge du partage d'IPD

  • PROTOCOLE D'ÉTUDE
  • SÈVE
  • CIF

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Non

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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