Clinical trial NCT06607302: Ivosidenib in Locally Advanced or Metastatic Cholangiocarcinoma With IDH1 R132 Mutation After at Least One Prior Systemic Treatment - an Observational Study
Official title: 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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cholangiocarcinoma
Lead sponsor: iOMEDICO AG
Sponsor class: INDUSTRY
Collaborators: name: Servier Deutschland GmbH
First posted: 2024-09-23
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Progression-free survival (PFS)
timeFrame: max. 38 months (FPI - LPLV)
Secondary outcomes:
measure: Overall survival (OS)
timeFrame: max. 38 months (FPI - LPLV)
measure: Timt to treatment failure (TTF)
timeFrame: max. 38 months (FPI - LPLV)
measure: Overall response rate (ORR)
timeFrame: max. 38 months (FPI - LPLV)
measure: Disease control rate (DCR)
timeFrame: max. 38 months (FPI - LPLV)
measure: (Serious) adverse events ((S)AE))
timeFrame: max. 38 months (FPI - LPLV)
measure: (Serious) adverse drug reactions ((S)ADR) related to ivosidenib
timeFrame: max. 38 months (FPI - LPLV)
measure: Adverse events of special interest (AESI)
timeFrame: max. 38 months (FPI - LPLV)
measure: Global health-related quality of life during course of treatment
timeFrame: max. 38 months (FPI - LPLV)
measure: Cholangiocarcinoma-related quality of life during course of treatment
timeFrame: max. 38 months (FPI - LPLV)
measure: Assessing parameters of physician treatment decision making
timeFrame: max. 30 months (recruitment period)
measure: Assessing parameters of physician treatment satisfaction
timeFrame: max. 32 months (recruitment period plus 8 weeks)
measure: Cumulative ivosidenib dose
timeFrame: max. 38 months (FPI - LPLV)
measure: Absolute dose intensity of ivosidenib
timeFrame: max. 38 months (FPI - LPLV)
measure: Relative dose intensity of ivosidenib
timeFrame: max. 38 months (FPI - LPLV)
measure: Frequency of dose modifications
timeFrame: max. 38 months (FPI - LPLV)
measure: Type of dose modifications
timeFrame: max. 38 months (FPI - LPLV)
measure: Reasons for dose modifications
timeFrame: max. 38 months (FPI - LPLV)
measure: Duration of treatment with ivosidenib
timeFrame: max. 38 months (FPI - LPLV)
measure: Reasons for end of treatment (EOT)
timeFrame: max. 38 months (FPI - LPLV)
measure: Type of last previous therapy
timeFrame: max. 38 months (FPI - LPLV)
measure: Frequency of last previous therapy
timeFrame: max. 38 months (FPI - LPLV)
measure: Duration of last previous therapy line
timeFrame: max. 38 months (FPI - LPLV)
measure: Concomitant medications
timeFrame: max. 38 months (FPI - LPLV)
measure: Concomitant medications known to induce QT prolongation
timeFrame: max. 38 months (FPI - LPLV)
measure: Subsequent antineoplastic therapies
timeFrame: max. 38 months (FPI - LPLV)
Other outcomes:
Locations:
city: Bad Mergentheim
country: Germany
facility: Caritas Krankenhaus Bad Mergentheim
city: Berlin
country: Germany
facility: Onkologisches Versorgungszentrum Berlin MVZ
Study officials:
References: