Biliary Tract Cancer
Conditions
Brief summary
This is a multicenter, non-randomized, umbrella, open-label phase II clinical study, aiming to observe and evaluate, as well as explore the efficacy and safety of precision targeted therapy based on NGS technology for IDH1-mutated patients, specifically the combination of ivosidenib with multi-target tyrosine kinase inhibitors represented by lenvatinib or PD-1/PD-L1 in advanced biliary tract cancer patients who have failed systemic chemotherapy.
Interventions
DRUGIvosidenib
Oral, selective, small-molecule inhibitor of the mutant isocitrate dehydrogenase 1 (IDH1) enzyme. Administered at a dose of 500 mg, taken orally once daily. This is the core investigational drug in all study arms.
DRUGLenvatinib
Oral, multi-targeted tyrosine kinase inhibitor. Administered at a weight-based dose (8 mg for body weight \<60 kg or 12 mg for body weight ≥60 kg), taken orally once daily. Used in combination arms.
BIOLOGICALPD-1/PD-L1 inhibitor
Intravenous immune checkpoint inhibitor. Specific agent (e.g., Pembrolizumab, Durvalumab, Toripalimab, or Tislelizumab) may be chosen based on local availability and patient access. Administered at standard doses (e.g., 200 mg, 1500 mg, or 240 mg) via IV infusion every three weeks. Used in combination arms.
Sponsors
Peking Union Medical College Hospital
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Voluntary Participation: Signed informed consent. * Genetic Mutation: Presence of an IDH1 mutation confirmed by genetic testing. * Disease Status： * Newly diagnosed, untreated advanced/metastatic disease; OR * Recurrence \>6 months after curative-intent surgery (with or without adjuvant therapy). * Measurable Disease: At least one measurable lesion per RECIST 1.1. * Performance Status: ECOG performance status of 0 or 1. * Life Expectancy： ≥3 months. * Organ Function: Adequate hematological, hepatic, and renal function. * Contraception: Use of highly effective contraception for women of childbearing potential and men.
Exclusion criteria
* Prior Treatment: Previous treatment with Ivosidenib. * Cancer Type: Ampulla of Vater cancer. * Pregnancy： Pregnant or breastfeeding women. * Allergy: Known hypersensitivity to any component of the study drugs. * Recent Therapy: Local anti-tumor therapy or major surgery within 4 weeks prior to initiation. * Medical Conditions: * Uncontrolled hypertension. * Significant cardiovascular disease. * Active or untreated CNS metastases. * Active autoimmune disease. * Uncontrolled active infection (e.g., HBV, HCV, HIV). * Significant bleeding tendency or history. * Severe non-healing wounds. * History of organ transplantation. * Concurrent Participation: Participation in another interventional clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Objective Response Rate (ORR) | From first dose of study drug until disease progression, death, or start of new anti-cancer therapy, assessed up to approximately 24 months. |
Secondary
| Measure | Time frame |
|---|---|
| Disease Control Rate (DCR) | From first dose of study drug until disease progression, death, or start of new anti-cancer therapy, assessed up to approximately 24 months. |
| Progression-Free Survival (PFS) | From first dose of study drug until disease progression or death from any cause (whichever occurs first), assessed up to approximately 24 months. |
| Overall Survival (OS) | From enrollment (or first dose) until death from any cause, assessed up to approximately 36 months. |
| Duration of Response (DOR) | From the date of first documented response (CR or PR) until the date of disease progression or death, assessed up to approximately 24 months. |
Countries
China
Contacts
Primary ContactChengjie Li
Outcome results
None listed