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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 juli 2026 bijgewerkt door: 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.

Studie Overzicht

Gedetailleerde beschrijving

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.

Studietype

Ingrijpend

Inschrijving (Geschat)

347

Fase

  • Fase 2

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Locaties

    • GUANGZHOU
      • Guangdong, GUANGZHOU, China, 510000

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

  • Volwassen
  • Oudere volwassene

Accepteert gezonde vrijwilligers

Nee

Beschrijving

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.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Actieve vergelijker: 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
Experimenteel: 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

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Tijdsspanne
Progression-Free Survival (PFS)
Tijdsspanne: Up to 2 Years
Up to 2 Years

Secundaire uitkomstmaten

Uitkomstmaat
Tijdsspanne
Responsduur (DOR)
Tijdsspanne: tot 2 jaar
tot 2 jaar
Ziektecontrolepercentage (DCR)
Tijdsspanne: tot 2 jaar
tot 2 jaar
Algehele overleving (OS)
Tijdsspanne: tot 2 jaar
tot 2 jaar
Objective Response Rate (ORR)
Tijdsspanne: Up to 2 Years
Up to 2 Years
quality of life score
Tijdsspanne: up to 2 years
up to 2 years
incidence and severity of adverse events (AEs)
Tijdsspanne: up to 2 years
up to 2 years
serious adverse events (SAEs)
Tijdsspanne: up to 2 years
up to 2 years

Andere uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Correlation analysis between TGFβ and Incidence of treatment response
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: up to 2 years
up to 2 years

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Geschat)

7 juli 2026

Primaire voltooiing (Geschat)

30 juni 2029

Studie voltooiing (Geschat)

30 juni 2029

Studieregistratiedata

Eerst ingediend

6 juli 2026

Eerst ingediend dat voldeed aan de QC-criteria

13 juli 2026

Eerst geplaatst (Werkelijk)

16 juli 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

16 juli 2026

Laatste update ingediend die voldeed aan QC-criteria

13 juli 2026

Laatst geverifieerd

1 juli 2026

Meer informatie

Termen gerelateerd aan deze studie

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

JA

Beschrijving IPD-plan

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

IPD-tijdsbestek voor delen

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

IPD-toegangscriteria voor delen

PI

IPD delen Ondersteunend informatietype

  • LEERPROTOCOOL
  • SAP
  • ICF

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Nee

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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