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HAIC + DEB-TACE + Toripalimab + Lenvatinib for Unresectable Intrahepatic Cholangiocarcinoma

28 juin 2026 mis à jour par: Shanghai Zhongshan Hospital

Hepatic Arterial Infusion Chemotherapy (HAIC) Sequential Small-Sized Drug-Eluting Beads Transarterial Chemoembolization (DEB-TACE) Combined With Toripalimab and Lenvatinib for Unresectable Intrahepatic Cholangiocarcinoma: A Phase II Clinical Study

Purpose: This phase II clinical trial evaluates whether a combination of liver-directed local therapies (HAIC and DEB-TACE) with immunotherapy (toripalimab) and targeted therapy (lenvatinib) is safe and effective for patients with unresectable intrahepatic cholangiocarcinoma (a type of liver cancer that cannot be removed by surgery).

Participants: Adults aged 18-85 years with pathologically confirmed unresectable intrahepatic cholangiocarcinoma, no prior immune checkpoint inhibitor therapy, and adequate organ function.

Study details include:

Study Duration: Up to 24 months per participant

Treatment Duration: Up to 6 cycles (each cycle is 21 days) of combination therapy, followed by maintenance therapy with toripalimab and lenvatinib until disease progression or unacceptable toxicity

Visit Frequency: Every 3 weeks during the treatment phase; tumor imaging assessments every 6-8 weeks

Primary endpoints: Objective response rate (ORR), progression-free survival (PFS), and overall survival (OS). Safety will be assessed by monitoring adverse events graded according to NCI-CTCAE v5.0.

Toripalimab and lenvatinib are not available through an expanded access program.

Aperçu de l'étude

Description détaillée

Background: Intrahepatic cholangiocarcinoma (ICC) is the second most common primary liver malignancy. Most patients are diagnosed at locally advanced or metastatic stages, precluding curative surgical resection. For unresectable ICC, conventional chemotherapy offers limited efficacy, with a 5-year survival rate below 10%. Emerging evidence suggests that combining locoregional therapies with immunotherapy and targeted therapy may improve outcomes.

Study Design: This is a prospective, single-arm, single-center, phase II exploratory clinical trial. Approximately 29 evaluable patients will be enrolled at Zhongshan Hospital, Fudan University.

Intervention: Participants will receive a comprehensive treatment regimen consisting of:

HAIC (Hepatic Arterial Infusion Chemotherapy): Oxaliplatin 85 mg/m² and gemcitabine (total 1000 mg/m², with a portion used for DEB-TACE loading) administered via hepatic artery infusion over ≥2 hours on Day 1 of each 21-day cycle, for up to 6 cycles.

DEB-TACE (Drug-Eluting Beads Transarterial Chemoembolization): Small-sized (40-90 μm) drug-eluting beads loaded with gemcitabine, performed on Day 1 of each cycle as needed (required in Cycle 1, thereafter based on tumor vascularity and imaging assessment).

Toripalimab: 200 mg intravenous infusion on Day 1 of each 21-day cycle.

Lenvatinib: Oral daily dosing (8 mg/day for body weight <60 kg; 12 mg/day for body weight ≥60 kg), continued throughout the study.

After completion of up to 6 cycles, patients without disease progression will enter a maintenance phase receiving toripalimab plus lenvatinib until disease progression, intolerable toxicity, withdrawal of consent, or investigator decision to terminate.

Efficacy Assessments: Tumor response will be evaluated by investigators using RECIST 1.1 and mRECIST criteria every 6-8 weeks. Primary efficacy endpoints include objective response rate (ORR), progression-free survival (PFS), and overall survival (OS).

Safety Monitoring: Adverse events will be monitored throughout the study and graded according to NCI-CTCAE v5.0. Safety visits will be conducted before each TACE procedure.

Statistical Considerations: The primary analysis will be descriptive. Sample size (approximately 29 patients) was calculated using a single-stage exact design based on an expected ORR of 40% versus a null rate of 15%, with α=0.05 and power=90%. Survival outcomes will be analyzed using Kaplan-Meier methods.

Type d'étude

Interventionnel

Inscription (Estimé)

29

Phase

  • Phase 2

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. Voluntary participation and signed informed consent.
  2. Age 18 to 85 years.
  3. Pathologically confirmed intrahepatic cholangiocarcinoma.
  4. Imaging-confirmed unresectable locally advanced intrahepatic cholangiocarcinoma with measurable lesions (longest diameter ≥10 mm) per RECIST 1.1.
  5. No distant organ metastases (excluding lymph node metastases).
  6. Child-Pugh liver function grade A or good B (≤7 points).
  7. ECOG performance status score 0-1 within 1 week before enrollment.
  8. Expected survival ≥12 weeks.
  9. No prior treatment with immune checkpoint inhibitors (including PD-1/PD-L1 antibodies and CTLA-4 inhibitors).
  10. Laboratory values within 7 days before enrollment meeting the following criteria:

ANC ≥1.0×10⁹/L; platelets ≥50×10⁹/L; hemoglobin ≥90 g/L (without transfusion or G-CSF within 14 days before screening).

Serum albumin ≥30 g/L; total bilirubin ≤1.5×ULN; ALT and AST ≤5×ULN; serum creatinine ≤1.5×ULN or CrCl >50 mL/min (Cockcroft-Gault formula).

INR ≤2.3 or PT prolonged ≤6 seconds above normal range.

Urine protein <2+ (if ≥2+, 24-hour quantification <1.0 g allowed).

Exclusion Criteria:

  1. Received other local treatments (excluding surgery) within 1 month before study entry. Prior TAE/TAI not allowed. Prior TACE >3 times not allowed.
  2. Prior systemic anti-tumor therapy (including targeted therapy, immunotherapy, chemotherapy).
  3. Concurrent or prior other malignancy within 5 years.
  4. Active autoimmune disease or history of autoimmune disease with potential relapse.
  5. Clinically symptomatic moderate-to-severe ascites requiring therapeutic paracentesis/drainage or Child-Pugh score >2; uncontrolled or moderate-to-large pleural/pericardial effusion.
  6. History of abdominal fistula, GI perforation, or intra-abdominal abscess within 6 months before study treatment.
  7. History of thrombosis or embolic events (e.g., cerebrovascular accident including TIA, cerebral hemorrhage, cerebral infarction, pulmonary embolism) within 6 months before study treatment.
  8. Known inherited or acquired bleeding disorder or thrombotic tendency; currently or recently (within 10 days) receiving full-dose anticoagulants or thrombolytics for therapeutic purposes (prophylactic low-dose aspirin or LMWH allowed).
  9. Major vascular disease within 6 months before study treatment (e.g., aortic aneurysm requiring surgical repair or recent peripheral arterial thrombosis).
  10. Severe, non-healing, or dehisced wounds, active ulcers, or untreated fractures.
  11. Major surgery within 4 weeks before study treatment (excluding diagnostic) or anticipated need for major surgery during the study.
  12. History of intestinal obstruction or clinical signs/symptoms of GI obstruction within 6 months before study treatment.
  13. History of hepatic encephalopathy.
  14. Palliative radiotherapy for non-target lesions allowed only if completed ≥2 weeks before study treatment and AEs recovered to ≤CTCAE grade 1.
  15. Severe infection within 4 weeks before study treatment, including hospitalization for infection, bacteremia, or severe pneumonia; oral or IV therapeutic antibiotics within 2 weeks (prophylactic allowed).
  16. Congenital or acquired immunodeficiency (e.g., HIV infection).
  17. Palliative radiotherapy involving >5% of bone marrow area within 4 weeks for patients with bone metastases.
  18. Received live attenuated vaccine within 28 days before study treatment, or expected to receive such vaccine during toripalimab treatment or within 60 days after last dose.
  19. Received other investigational drugs within 28 days before study treatment.
  20. Other factors judged by the investigator that may affect study results or cause premature termination, such as alcoholism, drug abuse, other serious diseases (including psychiatric) requiring concomitant treatment, severe laboratory abnormalities, family or social factors affecting patient safety.

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: N / A
  • Modèle interventionnel: Affectation à un seul groupe
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: HAIC + DEB-TACE + Toripalimab + Lenvatinib

Participants receive a combination regimen consisting of:

HAIC (Hepatic Arterial Infusion Chemotherapy): Oxaliplatin 85 mg/m² and gemcitabine (total 1000 mg/m², with a portion used for DEB-TACE loading) administered via hepatic artery infusion on Day 1 of each 21-day cycle, for up to 6 cycles.

DEB-TACE (Drug-Eluting Beads Transarterial Chemoembolization): Small-sized (40-90 μm) drug-eluting beads loaded with gemcitabine, performed on Day 1 of each cycle as needed (required in Cycle 1, thereafter based on tumor vascularity and imaging assessment).

Toripalimab: 200 mg intravenous infusion on Day 1 of each 21-day cycle.

Lenvatinib: Oral daily dosing (8 mg/day for body weight <60 kg; 12 mg/day for body weight ≥60 kg), continued throughout the study.

After completion of up to 6 cycles, patients without disease progression enter a maintenance phase receiving toripalimab plus lenvatinib until disease progression, intolerable toxicity, withdrawal of consent, or investigator decision to t

Small-sized (40-90 μm) drug-eluting beads loaded with gemcitabine, administered via transarterial chemoembolization into the hepatic artery to occlude tumor blood vessels and deliver localized chemotherapy. Performed on Day 1 of each 21-day cycle as needed (required in Cycle 1, thereafter based on tumor vascularity and imaging assessment).
200 mg administered as an intravenous infusion on Day 1 of each 21-day cycle. Toripalimab is a humanized anti-PD-1 monoclonal antibody that blocks PD-1/PD-L1 interaction, restoring T-cell anti-tumor immune activity.
Oral daily dosing: 8 mg/day for body weight <60 kg; 12 mg/day for body weight ≥60 kg. Lenvatinib is a multi-target tyrosine kinase inhibitor that inhibits VEGFR1-3, FGFR1-4, PDGFRα, KIT, and RET, thereby reducing tumor angiogenesis and suppressing tumor cell proliferation.
Total dose 1000 mg/m² administered via hepatic artery infusion (HAIC) on Day 1 of each 21-day cycle, for up to 6 cycles. A portion of the dose is used for DEB-TACE drug loading; the remainder is administered via HAIC. Gemcitabine is a pyrimidine nucleoside analog that inhibits DNA synthesis and induces tumor cell apoptosis.
85 mg/m² administered via hepatic artery infusion (HAIC) on Day 1 of each 21-day cycle, for up to 6 cycles. Oxaliplatin is a third-generation platinum-based chemotherapeutic agent that forms DNA crosslinks, blocking DNA replication and transcription, and inducing tumor cell apoptosis.

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Objective Response Rate (ORR)
Délai: From the start of treatment until disease progression, up to 24 months
Proportion of participants achieving a confirmed complete response (CR) or partial response (PR) according to RECIST 1.1 and mRECIST criteria, assessed by the investigator. This is the primary efficacy endpoint used for sample size calculation.
From the start of treatment until disease progression, up to 24 months
Progression-Free Survival (PFS)
Délai: From enrollment until disease progression or death, assessed up to 24 months
Time from enrollment to first documented disease progression per RECIST 1.1 and mRECIST criteria, or death from any cause, whichever occurs first.
From enrollment until disease progression or death, assessed up to 24 months
Overall Survival (OS)
Délai: From enrollment until death, assessed up to 24 months
Time from enrollment to death from any cause.
From enrollment until death, assessed up to 24 months

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Disease Control Rate (DCR)
Délai: From the start of treatment until disease progression, up to 24 months
Proportion of participants achieving a confirmed complete response (CR), partial response (PR), or stable disease (SD) according to RECIST 1.1 and mRECIST criteria, assessed by the investigator.
From the start of treatment until disease progression, up to 24 months
Duration of Response (DoR)
Délai: From first response until disease progression or death, assessed up to 24 months
Time from first documented response (CR or PR) to first documented disease progression per RECIST 1.1 and mRECIST criteria, or death from any cause, whichever occurs first.
From first response until disease progression or death, assessed up to 24 months
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Délai: From the time of informed consent until 30 days after the last dose of study treatment, or until resolution/return to baseline, assessed up to 24 months
Number of participants with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and laboratory abnormalities. Adverse events will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) Version 5.0.
From the time of informed consent until 30 days after the last dose of study treatment, or until resolution/return to baseline, assessed up to 24 months

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)

30 juin 2026

Achèvement primaire (Estimé)

30 juin 2028

Achèvement de l'étude (Estimé)

31 décembre 2028

Dates d'inscription aux études

Première soumission

28 juin 2026

Première soumission répondant aux critères de contrôle qualité

28 juin 2026

Première publication (Réel)

6 juillet 2026

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

6 juillet 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

28 juin 2026

Dernière vérification

1 juin 2026

Plus d'information

Termes liés à cette étude

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

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