Biliary Tract CancerPhase 2NCT07282262
An Exploratory Study on the Use of Ivosidenib for the Precise Treatment of Advanced Biliary Tract Malignancies With IDH1 Mutations in the Later Line of Therapy.
A Phase 2 study testing Ivosidenib, Lenvatinib and PD-1/PD-L1 inhibitor in people with Biliary Tract Cancer, run by Peking Union Medical College Hospital at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Biliary Tract Cancer
- What is being tested?
- Ivosidenib, Lenvatinib and PD-1/PD-L1 inhibitor
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 300 participants
- Where
- 1 siteChina
- Study dates
- 1 May 2024to 1 Oct 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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:
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Beijing Peking Union Medical College Hospital Outpatient Department
Beijing
Principal Investigator, +86-10-69152830, zhaoht@pumch.cn
About this study
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.
- Conditions
- Biliary Tract Cancer
- Being tested
- Ivosidenib, Lenvatinib, PD-1/PD-L1 inhibitor
- Medical subject headings
- Biliary Tract Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IvosidenibDrug
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.
LenvatinibDrug
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.
PD-1/PD-L1 inhibitorBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| Ivosidenib Monotherapy | Experimental | Not described Receives: Drug: Ivosidenib |
| Ivosidenib + Lenvatinib | Experimental | Not described Receives: Drug: Ivosidenib; Drug: Lenvatinib |
| Ivosidenib + PD-1/PD-L1 Inhibitor | Experimental | Not described Receives: Drug: Ivosidenib; Biological: PD-1/PD-L1 inhibitor |
| Ivosidenib + Lenvatinib + PD-1/PD-L1 Inhibitor | Experimental | Not described Receives: Drug: Ivosidenib; Drug: Lenvatinib; Biological: PD-1/PD-L1 inhibitor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR)
Measured: From first dose of study drug until disease progression, death, or start of new anti-cancer therapy, assessed up to approximately 24 months.
Secondary outcomes (4)
Disease Control Rate (DCR)
Measured: 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)
Measured: 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)
Measured: From enrollment (or first dose) until death from any cause, assessed up to approximately 36 months.
Duration of Response (DOR)
Measured: From the date of first documented response (CR or PR) until the date of disease progression or death, assessed up to approximately 24 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2025
- Study start1 May 2024
- Primary completion1 Oct 2026 (estimated)
- Study completion1 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Biliary Tract CancerStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07282262
- Sponsor's protocol ID
- NBLF001
- Status last verified
- Nov 2025
- First submitted
- 2 Dec 2025
- Last updated
- 15 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Study on the Use of Ivosidenib for the Precise Treatment of Advanced Biliary Tract Malignancies With IDH1 Mutations in the Later Line of Therapy.
- ClinicalTrials.gov ID
- NCT07282262
- Acronym / sponsor ID
- NBLF001
- Conditions
- Biliary Tract Cancer
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 1 May 2024 (actual)
- Primary completion
- 1 Oct 2026 (estimated)
- Study completion
- 1 Oct 2026 (estimated)
- First posted
- 15 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Ivosidenib Monotherapy | Experimental | Drug: Ivosidenib |
| Ivosidenib + Lenvatinib | Experimental | Drug: Ivosidenib, Drug: Lenvatinib |
| Ivosidenib + PD-1/PD-L1 Inhibitor | Experimental | Drug: Ivosidenib, Biological: PD-1/PD-L1 inhibitor |
| Ivosidenib + Lenvatinib + PD-1/PD-L1 Inhibitor | Experimental | Drug: Ivosidenib, Drug: Lenvatinib, Biological: PD-1/PD-L1 inhibitor |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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).
Key exclusion criteria (15 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IvosidenibOral, 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. | Drug | Ivosidenib + Lenvatinib, Ivosidenib + Lenvatinib + PD-1/PD-L1 Inhibitor, Ivosidenib + PD-1/PD-L1 Inhibitor, Ivosidenib Monotherapy |
| LenvatinibOral, 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. | Drug | Ivosidenib + Lenvatinib, Ivosidenib + Lenvatinib + PD-1/PD-L1 Inhibitor |
| PD-1/PD-L1 inhibitorIntravenous 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. | Biological | Ivosidenib + Lenvatinib + PD-1/PD-L1 Inhibitor, Ivosidenib + PD-1/PD-L1 Inhibitor |
Primary outcome
Objective Response Rate (ORR)
Time frame: From first dose of study drug until disease progression, death, or start of new anti-cancer therapy, assessed up to approximately 24 months.
4 secondary outcomes
Disease Control Rate (DCR)
Time frame: 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)
Time frame: 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)
Time frame: From enrollment (or first dose) until death from any cause, assessed up to approximately 36 months.
Duration of Response (DOR)
Time frame: From the date of first documented response (CR or PR) until the date of disease progression or death, assessed up to approximately 24 months.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ivosidenib, Lenvatinib and PD-1/PD-L1 inhibitor in people with Biliary Tract Cancer, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chengjie Li (13733879582, 1624271942@qq.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) (time frame: From first dose of study drug until disease progression, death, or start of new anti-cancer therapy, assessed up to approximately 24 months.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Dec 2025.