Cholangiocarcinoma Non-resectablePhase 2NCT06081829
A Phase 2 Study of Ivosidenib in Previously Treated Japanese Subjects With Nonresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation
A Phase 2 study testing Ivosidenib in people with Cholangiocarcinoma Non-resectable and Cholangiocarcinoma Metastatic, run by Servier at 7 sites in Japan.
Trial summary
This study will enroll participants with nonresectable or metastatic cholangiocarcinoma with an Isocitrate dehydrogenase protein, 1 (IDH1) mutation, who have previously received at least 1, but no more than 2, prior regimens for advanced disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cholangiocarcinoma Non-resectable, Cholangiocarcinoma Metastatic
- What is being tested?
- Ivosidenib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 7 sitesJapan
- Study dates
- 10 Oct 2023to May 2027
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have nonresectable or metastatic cholangiocarcinoma and are not eligible for curative resection, transplantation or ablative therapies
- Have documented IDH1 gene-mutated disease from a tumor biopsy
- Have an ECOG PS score of 0 or 1
- Have an expected survival of 3 months or more
- Have at least one evaluable and measurable lesion
- Have disease progression following the most recent of 1 or 2 prior systemic regimens for advanced disease with progression on the treatment that was most recently given at a minimum, and must have received at least 1 gemcitabine- or 5-FU -containing regimen
Show all 11Show fewer
- Have recovered from side effects associated with the prior treatment therapy
- Have adequate bone marrow function
- Have adequate hepatic (liver) and renal (kidney) function
- Women of child bearing potential must have a negative serum pregnancy test before starting study treatment, and use birth control during the study and for 90 days after the last dose of ivosidenib
- Fertile men with female partners of child bearing potential must use birth control during the study and for 90 days after the last dose of ivosidenib
Who cannot join
- Received a prior IDH inhibitor.
- Have known symptomatic brain metastases requiring steroids.
- Pregnancy, possibility of becoming pregnant during the study and breast-feeding women or woman who plans to restart breast-feeding after the study drug administration/intake.
- Are taking known strong cytochrome P450 (CYP) 3A4 inducers or sensitive CYP3A4 substrate medications with a narrow therapeutic window
- Have significant heart disease, including congestive heart failure, myocardial infarction (heart attack) unstable angina (chest pain) and/or stroke, within 6 months before starting the study
- Have a heart-rate corrected QT interval ≥450 msec or other factors that increase the risk of QT prolongation or arrhythmic events
- . Have active inflammatory gastrointestinal disease, chronic diarrhea, previous gastric resection or lap band dysphagia, short-gut syndrome, gastroparesis (paralysis of the stomach), or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally.
- Have known medical history of progressive multifocal leukoencephalopathy (PML)
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
Japan7 sites
National Cancer Center Hospital East (JPN-002)
Kashiwa, 277-8577
Kumamoto University Hospital (JPN-004)
Kumamoto, 860-8556
National Hospital Organization Shikoku Cancer Center (JPN-007)
Matsuyama, 791-0280
Osaka International Cancer Institute (JPN-005)
Osaka, 541-8567
Hokkaido University Hospital (JPN-006)
Sapporo, 060-8648
National Cancer Center Hospital (JPN-001)
Tokyo
Kanagawa Cancer Center (JPN-003)
Yokohama, 241-8515
About this study
Official title: A Phase 2, Open-label, Multicenter Study of Orally Administered Ivosidenib in Previously Treated Japanese Subjects With Nonresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation
This study will enroll participants with nonresectable or metastatic cholangiocarcinoma with an Isocitrate dehydrogenase protein, 1 (IDH1) mutation, who have previously received at least 1, but no more than 2, prior regimens for advanced disease. All participants will receive ivosidenib daily throughout multiple 28 day cycles. Study treatment will be administered until participant experiences unacceptable toxicity, disease progression, or other discontinuation criteria are met. Study visits will be conducted every week during Cycle 1 (Days 1, 8, 15, and 22), every other week during Cycles 2 and 3, and Day 1 of each cycle thereafter. After the last dose of treatment, participants will attend an end of treatment and a post-treatment follow-up visit, and participants will be followed to assess overall survival. Study visits may include a tumor assessment, physical exam, electrocardiogram (ECG), blood and urine analysis, and questionnaires.
- Being tested
- Ivosidenib
- Keywords
- IDH-1 Mutation, Cholangiocarcinoma Non-resectable with an IDH-1 Mutation, Cholangiocarcinoma Metastatic with an IDH-1 Mutation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IvosidenibDrug
Subjects will take 2 tablets (500 mg total) orally once daily.
| Study group | Role | What they receive |
|---|---|---|
| Open-Label Ivosidenib | Experimental | 250 mg Tablets Receives: Drug: Ivosidenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
6-month Progression Free Survival (PFS) Rate
Proportion of subjects who are alive and progression-free (using RECIST v1.1) at 6 months after Day 1 (C1D1) per Independent Radiology Center (IRC)
Measured: Through 6 months after the first dose
Secondary outcomes (20)
Progression Free Survival (PFS)
The time from Day 1 to the date of first documentation of disease progression as assessed by the Investigator and by the IRC per RECIST v1.1. or death due to any cause
Measured: Approximately 1 year
Overall Survival (OS)
Measured: Approximately 1 year
Objective Response (OR) Rate
Complete response or partial response
Measured: Approximately 1 year
Duration of Response (DOR)
The time from date of first documented confirmed complete response (CR) or confirmed partial response (PR) to date of first documented disease progression or death due to any cause
Measured: Approximately 1 year
Time to Response (TTR)
The time from Day 1 to date of first documented confirmed complete response (CR) or confirmed partial response (PR)
Measured: Approximately 1 year
Change From Baseline in Health-Related Quality of Life Using EORTC-QLQ-C30 Questionnaire Scores.
The European Organisation for Research and Treatment of Cancer - Quality Of Life Questionnaire - Core Questionnaire (EORTC-QLQ-C30) is comprised of 5 functional scales ((Physical functioning, Role functioning, Cognitive functioning, Emotional functioning and Social functioning), 3 symptom scales (Fatigue, Pain and Nausea/Vomiting), 6 additional single items (Dyspnoea, Insomnia, Appetite Loss, Constipation, Diarrhoea and Financial Difficulties) and global health status (GHS). All of the scale scores range from 0 - 100; for the functional scales and GHS the higher score represents better functioning and for the symptom scales and single items the higher score represents an increase in symptoms.
.
Measured: Baseline and 1 year
Change From Baseline in Health-Related Quality of Life Using EORTC-QLQ-BIL21 Questionnaire Scores.
The European Organisation for Research and Treatment of Cancer - Quality Of Life Questionnaire - Cholangiocarcinoma and Gallbladder Cancer Module (EORTC-QLQ-BIL21) scores range from 0 - 100 with higher scores representing more severe symptoms.
Measured: Baseline and 1 year
Average EQ-5D-5L VAS Scores
The 5-level EuroQol five dimensions questionnaire (EQ-5D-5L) visual analogue scale (VAS) scores range from 0 to 100 with a higher number representing a better health status.
Measured: Baseline, Cycle 3 Day 1 (cycle = 28 days), and End of Treatment Visit (within 5 to 33 days after last dose of treatment, approximately 1 year total)
Total Number of Adverse Events (AEs)
Measured: Approximately 1 year
Total Number of Participants With Adverse Events (AEs) Leading to Dose Modifications
Measured: Approximately 1 year
Total Number of Participants With Adverse Events (AEs) Leading to Discontinuation
Measured: Approximately 1 year
Total Number of Participants With Serious Adverse Events (SAEs)
Measured: Approximately 1 year
Total Number of Participants With Adverse Events (AEs) Leading to Death
Measured: Approximately 1 year
Average Area Under the Concentration-vs Time Curve From 0 to Time of Last Measurable Concentration (AUC0-t)
Measured: Cycle 1 Day 1 and Cycle 2 Day 1
Average AUC Over 1 Dosing Interval at Steady State (AUCtau,ss)
Measured: Cycle 2 Day 1
Average Time to Maximum Concentration (Tmax)
Measured: Cycle 1 Day 1 and Cycle 2 Day 1
Average Maximum Concentration (Cmax)
Measured: Cycle 1 Day 1 and Cycle 2 Day 1
Average Trough Concentration (Ctrough)
Measured: Cycle 2 Day 1
Average Plasma 2-hydroxyglutarate (2-HG) Concentrations
Measured: Cycle 1 Day 1 and Cycle 2 Day 1
Number of Participants With no Change, Plus 1 or Plus 2 Change From Baseline in Eastern Cooperative Oncology Group (ECOG) Performance Status (ECOG PS) Score
From baseline to worst value of post-baseline assessments. ECOG PS scores range from 0 to 5 with 0 representing a person being fully active and 5 being the patient is dead.
Measured: Approximately 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2023
- Study start10 Oct 2023
- Primary completion1 Oct 2024
- Study completionMay 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cholangiocarcinoma Non-resectableStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Servier
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06081829
- Sponsor's protocol ID
- CL2-95031-008
- Status last verified
- Nov 2025
- First submitted
- 14 Sep 2023
- Last updated
- 9 Dec 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data. Access can be requested for all interventional clinical studies: * used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US). * where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope. In addition, access can be requested for all interventional clinical studies in patients: * sponsored by Servier * with a first patient enrolled as of 1 January 2004 onwards * for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-label, Multicenter Study of Orally Administered Ivosidenib in Previously Treated Japanese Subjects With Nonresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation
- ClinicalTrials.gov ID
- NCT06081829
- Acronym / sponsor ID
- CL2-95031-008
- Keywords
- IDH-1 Mutation, Cholangiocarcinoma Non-resectable with an IDH-1 Mutation, Cholangiocarcinoma Metastatic with an IDH-1 Mutation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (actual)
- Lead sponsor
- ServierIndustry (pharma, biotech, devices)
- Study start
- 10 Oct 2023 (actual)
- Primary completion
- 1 Oct 2024 (actual)
- Study completion
- May 2027 (estimated)
- First posted
- 13 Oct 2023 (actual)
- Results
- Posted 9 Dec 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will enroll participants with nonresectable or metastatic cholangiocarcinoma with an Isocitrate dehydrogenase protein, 1 (IDH1) mutation, who have previously received at least 1, but no more than 2, prior regimens for advanced disease. All participants will receive ivosidenib daily throughout multiple 28 day cycles. Study treatment will be administered until participant experiences unacceptable toxicity, disease progression, or other discontinuation criteria are met. Study visits will be conducted every week during Cycle 1 (Days 1, 8, 15, and 22), every other week during Cycles 2 and 3, and Day 1 of each cycle thereafter. After the last dose of treatment, participants will attend an end of treatment and a post-treatment follow-up visit, and participants will be followed to assess overall survival. Study visits may include a tumor assessment, physical exam, electrocardiogram (ECG), blood and urine analysis, and questionnaires.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open-Label Ivosidenib | Experimental | Drug: Ivosidenib250 mg Tablets |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Have nonresectable or metastatic cholangiocarcinoma and are not eligible for curative resection, transplantation or ablative therapies
- Have documented IDH1 gene-mutated disease from a tumor biopsy
- Have an ECOG PS score of 0 or 1
- Have an expected survival of 3 months or more
- Have at least one evaluable and measurable lesion
Key exclusion criteria (8 listed)
- Received a prior IDH inhibitor.
- Have known symptomatic brain metastases requiring steroids.
- Pregnancy, possibility of becoming pregnant during the study and breast-feeding women or woman who plans to restart breast-feeding after the study drug administration/intake.
- Are taking known strong cytochrome P450 (CYP) 3A4 inducers or sensitive CYP3A4 substrate medications with a narrow therapeutic window
- Have significant heart disease, including congestive heart failure, myocardial infarction (heart attack) unstable angina (chest pain) and/or stroke, within 6 months before starting the study
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 |
|---|---|---|
| IvosidenibSubjects will take 2 tablets (500 mg total) orally once daily. | Drug | Open-Label Ivosidenib |
Primary outcome
6-month Progression Free Survival (PFS) Rate
Time frame: Through 6 months after the first dose
Proportion of subjects who are alive and progression-free (using RECIST v1.1) at 6 months after Day 1 (C1D1) per Independent Radiology Center (IRC)
20 secondary outcomes
Progression Free Survival (PFS)
Time frame: Approximately 1 year
Overall Survival (OS)
Time frame: Approximately 1 year
Objective Response (OR) Rate
Time frame: Approximately 1 year
Duration of Response (DOR)
Time frame: Approximately 1 year
Time to Response (TTR)
Time frame: Approximately 1 year
Change From Baseline in Health-Related Quality of Life Using EORTC-QLQ-C30 Questionnaire Scores.
Time frame: Baseline and 1 year
Change From Baseline in Health-Related Quality of Life Using EORTC-QLQ-BIL21 Questionnaire Scores.
Time frame: Baseline and 1 year
Average EQ-5D-5L VAS Scores
Time frame: Baseline, Cycle 3 Day 1 (cycle = 28 days), and End of Treatment Visit (within 5 to 33 days after last dose of treatment, approximately 1 year total)
Total Number of Adverse Events (AEs)
Time frame: Approximately 1 year
Total Number of Participants With Adverse Events (AEs) Leading to Dose Modifications
Time frame: Approximately 1 year
Total Number of Participants With Adverse Events (AEs) Leading to Discontinuation
Time frame: Approximately 1 year
Total Number of Participants With Serious Adverse Events (SAEs)
Time frame: Approximately 1 year
Total Number of Participants With Adverse Events (AEs) Leading to Death
Time frame: Approximately 1 year
Average Area Under the Concentration-vs Time Curve From 0 to Time of Last Measurable Concentration (AUC0-t)
Time frame: Cycle 1 Day 1 and Cycle 2 Day 1
Average AUC Over 1 Dosing Interval at Steady State (AUCtau,ss)
Time frame: Cycle 2 Day 1
Average Time to Maximum Concentration (Tmax)
Time frame: Cycle 1 Day 1 and Cycle 2 Day 1
Average Maximum Concentration (Cmax)
Time frame: Cycle 1 Day 1 and Cycle 2 Day 1
Average Trough Concentration (Ctrough)
Time frame: Cycle 2 Day 1
Average Plasma 2-hydroxyglutarate (2-HG) Concentrations
Time frame: Cycle 1 Day 1 and Cycle 2 Day 1
Number of Participants With no Change, Plus 1 or Plus 2 Change From Baseline in Eastern Cooperative Oncology Group (ECOG) Performance Status (ECOG PS) Score
Time frame: Approximately 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data. Access can be requested for all interventional clinical studies: * used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US). * where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope. In addition, access can be requested for all interventional clinical studies in patients: * sponsored by Servier * with a first patient enrolled as of 1 January 2004 onwards * for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
7 sites 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 in people with Cholangiocarcinoma Non-resectable and Cholangiocarcinoma Metastatic, run by Servier at 7 sites in Japan.
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 11 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 9 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 7 sites in Japan. 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?
Yes. Results are posted in the registry (first posted 9 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
6-month Progression Free Survival (PFS) Rate (time frame: Through 6 months after the first dose). 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 9 Dec 2025.