A Phase 2 Study of Ivosidenib in Previously Treated Japanese Subjects With Nonresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation

NCT ID: NCT06081829

Last Updated: 2025-12-09

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-10-10
• Study Completion Date: 2027-05-31

Brief 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. 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.

Conditions

Cholangiocarcinoma Non-resectable; Cholangiocarcinoma Metastatic

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Open-Label Ivosidenib

250 mg Tablets

Group Type: EXPERIMENTAL

Ivosidenib

Intervention Type: DRUG

Subjects will take 2 tablets (500 mg total) orally once daily.

Interventions

Ivosidenib

Subjects will take 2 tablets (500 mg total) orally once daily.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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
• 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

Exclusion Criteria

• 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)

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Servier

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

National Cancer Center Hospital East (JPN-002)

Kashiwa, , Japan

Kumamoto University Hospital (JPN-004)

Kumamoto, , Japan

National Hospital Organization Shikoku Cancer Center (JPN-007)

Matsuyama, , Japan

Osaka International Cancer Institute (JPN-005)

Osaka, , Japan

Hokkaido University Hospital (JPN-006)

Sapporo, , Japan

National Cancer Center Hospital (JPN-001)

Tokyo, , Japan

Kanagawa Cancer Center (JPN-003)

Yokohama, , Japan

Countries

Japan

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

CL2-95031-008

Identifier Type: -

Identifier Source: org_study_id