Ivosidenib in Locally Advanced or Metastatic Cholangiocarcinoma With IDH1 R132 Mutation After at Least One Prior Systemic Treatment - an Observational Study (IDHIRA)

November 25, 2025 updated by: iOMEDICO AG

Ivosidenib in Locally Advanced or Metastatic Cholangiocarcinoma With IDH1 R132 Mutation After at Least One Prior Systemic Treatment - a Prospective, Multicenter, Observational Study in Germany

Cholangiocarcinoma is a rare and aggressive tumor of the bile duct associated with a poor prognosis and very limited treatment options. The IDH1 inhibitor ivosidenib provides a new, targeted treatment option for this disease. Ivosidenib was approved by European Medicines Agency (EMA) in May 2023 as monotherapy in adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy.

The prospective, multicenter, observational study IDHIRA will collect first real-world data on ivosidenib treatment in a broad patient population in Germany. Ivosidenib will be administered according to the current SmPC. Thus, IDHIRA will generate real-world evidence on effectiveness, quality of life (QoL) and safety of ivosidenib.

Study Overview

Status

Recruiting

Conditions

Study Type

Observational

Enrollment (Estimated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Germany
      • Bad Liebenwerda, Germany
        • Recruiting
        • Hämatologisch-Onkologische Schwerpunktpraxis in Bad Liebenwerda
        • Contact:
          • Stephan Kreher, PD Dr. med.
      • Bad Mergentheim, Germany
        • Recruiting
        • Caritas Krankenhaus Bad Mergentheim
        • Contact:
          • Matthias Raab, Dr.
      • Berlin, Germany
        • Recruiting
        • Onkologisches Versorgungszentrum Berlin MVZ
        • Contact:
          • Reinhard Musch
      • Hanover, Germany
        • Recruiting
        • Onkologie Hannover
        • Contact:
          • Haytham Kamal, Dr. med.
      • Hof, Germany
        • Recruiting
        • Onkologie Hof
        • Contact:
          • Markus Kapp, Dr. med.
      • Mönchengladbach, Germany
        • Recruiting
        • Medizinisches Versorgungszentrum Mönchengladbach
        • Contact:
          • Ludger Sellmann, PD Dr. med.
      • Weinheim, Germany
        • Recruiting
        • ze:roPRAXEN MVZ für Innere Medizin
        • Contact:
          • Manuel Zink, Dr. med.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients with IDH1 R132-mutated locally advanced or metastatic CCC who have received at least one prior line of systemic therapy and who have already been scheduled by their treating physician to receive ivosidenib prior to enrolment in IDHIRA are eligible to participate.

Description

Inclusion Criteria:

  • Age 18 years or older.
  • Histologically confirmed locally advanced or metastatic CCC with a documented IDH1 R132 mutation diagnosed by an appropriate diagnostic test
  • Patients must have at least one prior systemic therapy
  • Decision for treatment with ivosidenib according to current SmPC.
  • Signed written informed consent before or within 6 weeks of first ivosidenib dose (inclusion of patients up to 6 weeks after first ivosidenib intake is allowed for patients not participating in the PRO module)

  • For patients participating in the PRO module (optional):

    • Dated signature of informed consent form before start of study treatment.
    • Willingness and capability to participate in PRO assessment in German language.
  • Other criteria according to current SmPC.

Exclusion Criteria:

  • Participation in an interventional clinical trial within 30 days prior to enrolment or concurrent participation in an interventional clinical trial except for the follow-up period.
  • Other contraindications according to current SmPC.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Progression-free survival (PFS)
Time Frame: max. 38 months (FPI - LPLV)
PFS is defined as the time interval measured from the day of first ivosidenib administration to first progression or death, whichever comes first. Patients without tumor progression or death at the time of analysis will be censored at their date of last contact.
max. 38 months (FPI - LPLV)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall survival (OS)
Time Frame: max. 38 months (FPI - LPLV)
OS is defined as the time interval measured from the day of first ivosidenib administration to time of death from any cause. Time to last contact will be used if a patient has no documented date of death and OS for the patient will be considered censored.
max. 38 months (FPI - LPLV)
Timt to treatment failure (TTF)
Time Frame: max. 38 months (FPI - LPLV)
TTF is defined as the time interval measured from the day of first ivosidenib until discontinuation of treatment for any reason including progression, toxicity, start of a new antineoplastic therapy, or death, whichever occurs first. Patients dropping out without knowledge of a potential ending of therapy (e.g., lost to follow up) will be censored with the last date of contact.
max. 38 months (FPI - LPLV)
Overall response rate (ORR)
Time Frame: max. 38 months (FPI - LPLV)
ORR is defined as the proportion of patients achieving a complete or partial response as best response. Patients without response measurement are considered non-responders.
max. 38 months (FPI - LPLV)
Disease control rate (DCR)
Time Frame: max. 38 months (FPI - LPLV)
DCR is defined as the proportion of patients achieving complete response, partial response, or stable disease as best response. Patients without response measurement are considered non-responders.
max. 38 months (FPI - LPLV)
(Serious) adverse events ((S)AE))
Time Frame: max. 38 months (FPI - LPLV)
The case- and patient-based incidence of (S)AEs will be provided.
max. 38 months (FPI - LPLV)
(Serious) adverse drug reactions ((S)ADR) related to ivosidenib
Time Frame: max. 38 months (FPI - LPLV)
The case- and patient-based incidence of (S)ADRs will be provided.
max. 38 months (FPI - LPLV)
Adverse events of special interest (AESI)
Time Frame: max. 38 months (FPI - LPLV)
The case- and patient-based incidence of AESIs will be provided.
max. 38 months (FPI - LPLV)
Assessing parameters of physician treatment decision making
Time Frame: max. 30 months (recruitment period)
Frequencies of distinct impact ratings for parameters affecting ivosidenib therapy choice will be visualized using stacked bar charts.
max. 30 months (recruitment period)
Assessing parameters of physician treatment satisfaction
Time Frame: max. 32 months (recruitment period plus 8 weeks)
Frequencies of distinct satisfaction levels with ivosidenib treatment effectiveness and AE management will be visualized using stacked bar charts.
max. 32 months (recruitment period plus 8 weeks)
Cumulative ivosidenib dose
Time Frame: max. 38 months (FPI - LPLV)
Summary tables containing descriptive statistics (n, mean, standard deviation, median, 25th and 75th percentiles, minimum, and maximum) will be provided.
max. 38 months (FPI - LPLV)
Absolute dose intensity of ivosidenib
Time Frame: max. 38 months (FPI - LPLV)
Summary tables containing descriptive statistics (n, mean, standard deviation, median, 25th and 75th percentiles, minimum, and maximum) will be provided.
max. 38 months (FPI - LPLV)
Relative dose intensity of ivosidenib
Time Frame: max. 38 months (FPI - LPLV)
Summary tables containing descriptive statistics (n, mean, standard deviation, median, 25th and 75th percentiles, minimum, and maximum) will be provided.
max. 38 months (FPI - LPLV)
Frequency of dose modifications
Time Frame: max. 38 months (FPI - LPLV)
Frequency of dose modifications will be presented.
max. 38 months (FPI - LPLV)
Type of dose modifications
Time Frame: max. 38 months (FPI - LPLV)
Type of dose modifications (i.e., dose reductions, dose escalations, and interruptions) will be presented.
max. 38 months (FPI - LPLV)
Reasons for dose modifications
Time Frame: max. 38 months (FPI - LPLV)
Reasons for dose modifications will be presented.
max. 38 months (FPI - LPLV)
Duration of treatment with ivosidenib
Time Frame: max. 38 months (FPI - LPLV)
Duration of treatment with ivosidenib
max. 38 months (FPI - LPLV)
Reasons for end of treatment (EOT)
Time Frame: max. 38 months (FPI - LPLV)
Reasons for end of treatment will be displayed
max. 38 months (FPI - LPLV)
Type of last previous therapy
Time Frame: max. 38 months (FPI - LPLV)
Type of substances given in the last previous therapy will be displayed.
max. 38 months (FPI - LPLV)
Frequency of last previous therapy
Time Frame: max. 38 months (FPI - LPLV)
Frequency of different substances given in the last previous therapy will be displayed.
max. 38 months (FPI - LPLV)
Duration of last previous therapy line
Time Frame: max. 38 months (FPI - LPLV)
Duration of last previous therapy line will be displayed.
max. 38 months (FPI - LPLV)
Concomitant medications
Time Frame: max. 38 months (FPI - LPLV)
Frequency of concomitant medications in total will be displayed.
max. 38 months (FPI - LPLV)
Concomitant medications known to induce QT prolongation
Time Frame: max. 38 months (FPI - LPLV)
Frequency of concomitant medications known to induce QT prolongation (e.g., antiarrhythmic medicines, fluoroquinolones, triazole anti-fungals, 5-HT3 receptor antagonists) will be displayed.
max. 38 months (FPI - LPLV)
Subsequent antineoplastic therapies
Time Frame: max. 38 months (FPI - LPLV)
Frequency and type of subsequent antineoplastic therapies by line of therapy will be displayed.
max. 38 months (FPI - LPLV)
Global health-related quality of life during course of treatment
Time Frame: max. 38 months (FPI - LPLV)

The change from baseline (i.e., difference) in the scales of the EORTC QLQ-C30 (European Organisation for Research and Treatment of Cancer quality of life questionnaire C30) will be displayed for each point in time, using boxplots.

The scales of the EORTC QLQ-C30 range in score from 0 to 100. A high scale score represents a higher response level.
max. 38 months (FPI - LPLV)
Cholangiocarcinoma-related quality of life during course of treatment
Time Frame: max. 38 months (FPI - LPLV)

The change from baseline (i.e., difference) in the scales of the EORTC QLQ-BIL21 (European Organisation for Research and Treatment of Cancer quality of life questionnaire BIL21) will be displayed for each point in time, using boxplots.

The scales of the EORTC QLQ-BIL21 range in score from 0 to 100. A high scale score represents a higher response level.
max. 38 months (FPI - LPLV)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

iOMEDICO AG

Collaborators

Servier Deutschland GmbH

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 8, 2024

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

December 1, 2027

Study Registration Dates

First Submitted

September 16, 2024

First Submitted That Met QC Criteria

September 18, 2024

First Posted (Actual)

September 23, 2024

Study Record Updates

Last Update Posted (Actual)

November 26, 2025

Last Update Submitted That Met QC Criteria

November 25, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • iOM-040498

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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