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.
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.
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.
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.
Cohort
Prospective
Not listed
Progression-free survival (PFS)
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.
Overall survival (OS)
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.
Timt to treatment failure (TTF)
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.
Overall response rate (ORR)
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.
Disease control rate (DCR)
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.
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iOMEDICO AG
Lead sponsor
Servier Deutschland GmbH
Collaborator
