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

NCT06081829 | Active Not Recruiting Phase 2 Results FDA Drug

• 1 other identifier: CL2-95031-008
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 7

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

Keywords

IDH-1 Mutation; Cholangiocarcinoma Non-resectable with an IDH-1 Mutation; Cholangiocarcinoma Metastatic with an IDH-1 Mutation

Outcome Measures

Primary Outcomes (1)

• 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)

  Through 6 months after the first dose

Secondary Outcomes (20)

• Progression Free Survival (PFS)

  Approximately 1 year

• Overall Survival (OS)

  Approximately 1 year

• Objective Response (OR) Rate

  Approximately 1 year

• Duration of Response (DOR)

  Approximately 1 year

• Time to Response (TTR)

  Approximately 1 year

Study Arms (1)

Open-Label Ivosidenib

EXPERIMENTAL

250 mg Tablets

Drug: Ivosidenib

Interventions

Ivosidenib DRUG

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

Open-Label Ivosidenib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Servier: lead

Study Sites (7)

National Cancer Center Hospital East (JPN-002)

Kashiwa, 277-8577, Japan

Location

Kumamoto University Hospital (JPN-004)

Kumamoto, 860-8556, Japan

Location

National Hospital Organization Shikoku Cancer Center (JPN-007)

Matsuyama, 791-0280, Japan

Location

Osaka International Cancer Institute (JPN-005)

Osaka, 541-8567, Japan

Location

Hokkaido University Hospital (JPN-006)

Sapporo, 060-8648, Japan

Location

National Cancer Center Hospital (JPN-001)

Tokyo, Japan

Location

Kanagawa Cancer Center (JPN-003)

Yokohama, 241-8515, Japan

Location

MeSH Terms

Interventions

ivosidenib

Results Point of Contact

• Title: Clinical Studies Department
• Organization: Institut de Recherches Internationales Servier (I.R.I.S.)

scientificinformation@servier.com

+33 1 55 72 60 00

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 14, 2023
• First Posted: October 13, 2023
• Study Start: October 10, 2023
• Primary Completion: October 1, 2024
• Study Completion (Estimated): May 1, 2027
• Last Updated: December 9, 2025
• Results First Posted: December 9, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: After Marketing Authorization in EEA or US if the study is used for the approval.
• Access Criteria: Researchers should register on Servier Data Portal and fill in the research proposal form. This form in four parts should be fully documented. The Research Proposal Form will not be reviewed until all mandatory fields are completed.

Locations

JP (7)