The purpose of this study is to learn more about the safety and efficacy of ivosidenib taken with azacitidine to treat adult patients with acute myeloid leukemia (AML) who are presenting a gene mutation called IDH1 (isocitrate dehydrogenase1 mutation-positive \[IDH1m\]) and cannot receive treatment with intensive chemotherapy (IC).
Participants who are eligible and enroll in the study will attend a study visit on the first day of each 28-day cycle. Study visits will consist of a physical exam, blood work, electrocardiogram (ECG) and other assessments. After treatment discontinuation participants will be contacted every 12 weeks through the end of the study (currently planned for 2026) to assess survival. The study drug, Ivosidenib, will be taken once daily throughout the duration of participation in the study, and Azacitidine will only be administered for 7 days at the beginning of each 28 day cycle. If at any point ivosidenib is made available as a medical prescription at the patient's site, the patient will switch to commercial product and will continue to be followed according to the protocol.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
245
Provided as tablets, taken orally as two 250mg tablets once daily.
Administered subcutaneously (SC) or intravenously (IV) at a dose of 75mg/m2/day for 7 days, either consecutively on Days 1-7 or discontinuously for Days 1-5 and 8-9 of each cycle. The 7 days of administration will occur at the beginning of every 4 week-long cycle.
AKH - Medizinische Universität Wien
Vienna, Austria
COMPLETEDKlinikum Wels-Grieskirchen GmbH
Wels, Austria
COMPLETEDInstitut Paoli Calmettes
Marseille, Bouches-du-Rhône, France
RECRUITINGCHU CAEN - Hôpital de la Côte de Nacre
Caen, Calvados, France
RECRUITINGCHU de Toulouse pt
Toulouse, Haute Garonne, France
RECRUITINGCHU Rennes - Hopital Pontchaillou
Rennes, Ille Et Vilaine, France
NOT_YET_RECRUITINGCHU Angers - Hôpital Hôtel Dieu
Angers, Liore, France
NOT_YET_RECRUITINGIRCCS Istituto Scientifico Romagnolo Per Lo Studio Dei Tumori "Dino Amadori" - IRST
Meldola, Forli-Cesena, Italy
RECRUITINGAzienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (Presidio Spedali Civili)
Brescia, Italy
RECRUITINGIRCCS Ospedale Policlinico San Martino
Genova, Italy
RECRUITING...and 5 more locations
Number of Adverse Events (AEs)
Adverse events (AEs) will be graded according to the CTCAE v5.0
Time frame: up to week 116
Number of Serious Adverse Events (SAEs)
Adverse events (AEs) will be graded according to the CTCAE v5.0
Time frame: up to week 116
Differentiation Syndrome of Grade 2 or higher
Time frame: up to week 116
Number of Adverse Events (AEs) leading to ivosidenib + azacitidine discontinuation
Time frame: up to week 112
Number of Adverse Events (AEs) leading to ivosidenib + azacitidine interruption
Time frame: up to week 112
Number of Adverse Events (AEs) leading to ivosidenib + azacitidine dose reduction
Time frame: up to week 112
Number of Adverse Events (AEs) leading to death
Time frame: up to week 116
Number of clinical laboratory anomalies assessed as Adverse Events (AEs)
Time frame: up to week 116
Number of patients requiring transfusion (platelet and RBC) and the average number of units transfused
Time frame: up to week 116
Rate of infections
Infection rates will be summarized by classification and will include a count and proportion.
Time frame: up to week 116
QT Prolongation event assessed as Grade 3 or higher
Time frame: up to week 116
Event-free survival (EFS)
Time frame: up to week 116
Proportion of patients who achieve a complete remission (CR)
Time frame: up to week 116
Proportion of patients who achieve complete remission plus complete remission with partial hematologic recovery rate (CR + CRh)
Time frame: up to week 116
Proportion of patients who achieve complete remission plus complete remission with incomplete hematologic recovery rate (CR + CRi)
Time frame: up to week 116
Duration of response (DOR)
Time frame: up to week 116
Time to response (TTR)
Time frame: up to week 116
Overall survival (OS)
Time frame: until study closure
Quality of life (QoL), as measured by Hematologic Malignancy-Patient-Reported Outcome (HM-PRO)
For patients with a baseline assessment and at least 1 post-baseline assessment that generates a score
Time frame: up to week 116
Quality of life (QoL), as measured by Family Reported Outcome Measure (FROM-16), for caregivers and/or family
For patients with a baseline assessment and at least 1 post-baseline assessment that generates a score
Time frame: up to week 116
Health economic measures, as assessed by the 5-level EuroQol 5-Dimensions (EQ-5D-5L)
For patients with a baseline assessment and at least 1 post-baseline assessment that generate a score
Time frame: up to week 116
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Average proportion of days at home
Defined by subtracting the number of care days (days hospitalized or seen in an ED / oncology clinic / infusion center) from the total days of follow-up, divided by total days of follow-up
Time frame: up to week 116