Ivosidenib (TIBSOVO®) Combined With Azacitidine According to Current SmPC (CONFIDHENCE)

Ivosidenib in Combination With Azacitidine as First-line Treatment for Adult Patients With Newly Diagnosed AML With an IDH1 R132 Mutation Who Are Not Eligible to Receive Standard Induction Chemotherapy

The goal of this non-interventional study is to evaluate quality of life (QoL) in adult patients with newly diagnosed IDH1 R132-mutated AML who are not eligible to receive standard induction chemotherapy and who are treated with ivosidenib in combination with azacitidine in a real-world setting in Germany.

The main questions it aims to answer are:

• Evaluate QoL by validated and widely used Functional Assessment of Cancer Therapy - Leukemia (FACT-Leu) questionnaire and European Quality of Life 5 Dimensions 5 Level Version (EQ-5D-5L) questionnaire during treatment and follow-up period
• Assesment of effectiveness in routine treatment (e.g. overall survival, event-free survival, overall response rate)
• Assessment of drug safety (all adverse events)
• Description of treatment reality in detail

Study Overview

• Status: Completed
• Conditions: Acute Myeloid Leukemia (AML)
• Intervention / Treatment: Drug: Ivosidenib

• Study Type: Observational
• Enrollment (Actual): 8

Contacts and Locations

Study Locations

• Germany
  • Freiburg im Breisgau, Germany
    • Praxis für interdisziplinäre Onkologie & Hämatologie

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult patients with newly diagnosed, IDH1 R132- mutated AML who are not eligible to receive standard induction chemotherapy and with treatment decision for ivosidenib in combination with azacitidine according to SmPC.

Description

Inclusion Criteria:

• Age 18 years or older.
• Newly diagnosed Acute Myeloid Leukemia (AML).
• Having an isocitrate dehydrogenase 1 (IDH1) R132 mutation.
• Not eligible to receive standard induction chemotherapy.
• Decision for treatment with ivosidenib in combination with azacitidine as per current SmPC.

• Signed written informed consent*

  *Patients are allowed to be enrolled up to 6 weeks after ivosidenib plus azacitidine dose

• For patients participating in PROs: 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 enrollment or simultaneous participation in an interventional clinical trial except for the follow-up period.
• Patients unable to consent
• Other contraindications according to current SmPC.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluate quality of life	Validation of FACT-Leu. Change from baseline (treatment start) of FACT-Leu total score over time	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assessment of parameters of treatment decision making	Frequency of distinct parameters affecting therapy choice including reasons why patient is ineligible for standard induction chemotherapy.	From date of patient enrollment until start of treatment
Subjective well-being: Validation of FACT-Leu	Change from baseline (treatment start) of FACT-Leu subscale score over time	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of FACT-Leu	Change from baseline (treatment start) of FACT-G total scores over time	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of FACT-Leu	Change from baseline (treatment start) of Trial outcome Index over time	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of FACT-Leu	Time to deterioration of FACT-Leu total score	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of FACT-Leu	Time to deterioration of FACT-G total score	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of FACT-Leu	Time to deterioration of Trial Outcome Index	Baseline until end of study (during ivosidinib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of FACT-Leu and EQ-5D-5L questionnaire	FACT-Leu total score over time over time	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of FACT-Leu and EQ-5D-5L questionnaire	FACT-G total score over time	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of FACT-Leu and EQ-5D-5L questionnaire	Trial Outcome Index	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of EQ-5D-5L questionnaire	Change from baseline of the EQ-5D-5L visual analogue scale score over time	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of EQ-5D-5L questionnaire	EQ-5D-5L visual analogue scale score over time	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of EQ-5D-5L questionnaire	Index value of EQ-5D-5L over time	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Subjective well-being: Validation of EQ-5D-5L questionnaire	Time to deterioration of the EQ-%D-5l visual analogue scale score over time	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Assesment of effectiveness in routine treatment	Assesment of overall survival (OS),	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Assesment of effectiveness in routine treatment	Event-free survival (EFS)	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Assesment of effectiveness in routine treatment	Overall Response Rate (ORR; i.e. CR, CR with incomplete hematologic recovery (Cri), (including CR with incomplete platelet recovery (CRp), CR with partial hematologic recovery (CRh), or partial recovery (PR)), Duration of CR (DOCR), Duration of Response (DOR; CR, Cri, CRp, CRh, PR))	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Assesment of effectiveness in routine treatment	Time to first response (TTR)	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Assessment of drug safety	Assesment of adverse events (AEs), serious adverse events (SAEs), adverse drug reactions (ADRs), serious adverse drug reactions (SADRs), Adverse events of special interest (AESIs)	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Ivosidenib and azacitidine treatment: Dose intensity	Descriptive statistics will be provided for dose intensity for ivosidinib and azacitidine	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Ivosidenib and azacitidine treatment: Frequency and type of dose modification	Frequency tables will be provided for frequency and type of dose modification for ivosidinib and azacitidine	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Ivosidenib and azacitidine treatment: Reason for dose modifications	Frequency tables of reasons for dose modifications will be provided for ivosidinib and azacitidine	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Ivosidenib and azacitidine treatment: Duration of treatment in total and for each substance	Descriptive statistics will be provided for duration of treatment in total and for each substance dose	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Ivosidenib and azacitidine treatment: Reason for end of treatment (EOT)	Frequency tables of EOT-reasons will be provided	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Treatment reality in detail: Transfusion dependency	Proportion of transfusion dependent patients to independency and vice versa will be provided	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Treatment reality in detail: Concomitant medication	Frequency table of concomitant medication in total and frequency of concomitant medications known to induce QT prolongation (e.g., antiarrhythmic medicines, fluoroquinolones, triazole anti-fungals, 5-HT3 receptor antagonists) as well as strong CYP3A4 inducers or dabigatran will be provided	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Treatment reality in detail: Subsequent antineoplastic therapies	Description of frequency and type of subsequent antineoplastic therapies by line of therapy (number and substance)	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Treatment reality in detail: Frequency of hospitalizations/emergency room visits	Frequency table of hospitalization and emergency room visits will be provided	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Treatment reality in detail: Reasons for hospitalizations/emergency room visits	Frequency table of reasons for hospitalization and emergency room visits will be provided	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months
Treatment reality in detail: Length of hospital stay	Descriptive statistic for length of hospital stay will be provided	Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months

Collaborators and Investigators

• Sponsor: iOMEDICO AG

Study record dates

Study Major Dates

• Study Start (Actual): December 20, 2023
• Primary Completion (Actual): July 30, 2025
• Study Completion (Actual): July 30, 2025

Study Registration Dates

• First Submitted: November 27, 2023
• First Submitted That Met QC Criteria: December 12, 2023
• First Posted (Actual): December 26, 2023

Study Record Updates

• Last Update Posted (Actual): February 10, 2026
• Last Update Submitted That Met QC Criteria: February 6, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: IDH1 R132 mutation
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Leukemia; Hemic and Lymphatic Diseases; Leukemia, Myeloid, Acute; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; ivosidenib
• Other Study ID Numbers: iOM-070496

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No