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Brief description/objective

IDH1 or IDH2 mutations are each found in about 10% of AML – and specific inhibitors are approved or in development in this indication.
The primary objective of this study is to compare event-free survival (EFS) between ivosidenib/enasidenib and placebo in combination with induction therapy and consolidation therapy followed by maintenance therapy in patients with newly diagnosed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) with excess blasts-2 (EB2), with an IDH1 or IDH2 mutation, eligible for intensive chemotherapy.

Key secondary objective:
To determine if treatment including ivosidenib/enasidenib, as compared to placebo, prolongs overall survival (OS).

Other secondary objectives:
To compare relapse-free survival (RFS), cumulative incidence of relapse (CIR) and cumulative incidence of death (CID) after complete response (CR) and CR with incomplete hematologic recovery (CRi) between treatment including ivosidenib/enasidenib and treatment including placebo.
To evaluate minimal residual disease (MRD) status at sequential time points throughout treatment and CRMRD− rates between treatment including ivosidenib/enasidenib vs. placebo, using molecular and/or flow cytometric techniques.
To assess the safety and tolerability of treatment including ivosidenib/enasidenib vs. placebo.
To compare CR/CRi rates for treatment including ivosidenib/enasidenib vs. placebo.
To assess the time to hematopoietic recovery (ANC 0.5 and 1.0x109/l; platelets 50 and 100x109/l) after each chemotherapy treatment cycle.
To determine quality of life (QoL) during maintenance treatment with ivosidenib/enasidenib vs. placebo.
Exploratory objective:
To study the pharmacokinetics of treatment including ivosidenib/enasidenib in a small subset of patients