Efficacy and Safety Profile of Ivosidenib in the Management of Patients with Acute Myeloid Leukemia (AML): An Update on the Emerging Evidence

Figures & data

Table 1 Current Clinical Trials in AML and MDS

NCT Number	Title	Conditions	Interventions	Estimated Patient Enrollment	Characteristics
NCT04493164	CPX-351 and ivosidenib for the treatment of IDH1 mutated acute myeloid leukemia or high-risk myelodysplastic syndrome	• Acute myeloid leukemia with gene mutations • Myelodysplastic syndrome • Myeloproliferative neoplasm • Recurrent/Refractory acute myeloid leukemia	• Drug: Ivosidenib • Drug: Liposome-encapsulated daunorubicin–cytarabine	30	Phase 2
NCT04250051	Ivosidenib and combination chemotherapy for the treatment of IDH1 mutant relapsed or refractory acute myeloid leukemia	• Recurrent/Refractory acute myeloid leukemia • Recurrent/Refractory myelodysplastic syndrome • Recurrent Myeloproliferative neoplasm	• Drug: Cytarabine • Biological: Filgrastim • Drug: Fludarabine • Drug: Fludarabine Phosphate • Drug: Ivosidenib	25	Phase 1
NCT04176393	A China bridging study of ivosidenib in r/r AML subjects with an IDH1 mutation	• Relapsed/Refractory acute myeloid leukemia	• Drug: Ivosidenib	30	Phase 1
NCT03839771	A study of ivosidenib or enasidenib in combination with induction therapy and consolidation therapy, followed by maintenance therapy in patients with newly diagnosed acute myeloid leukemia or myedysplastic syndrome EB2, with an IDH1 or IDH2 mutation, respectively, eligible for intensive chemotherapy	• Acute myeloid leukemia • Myelodysplastic syndrome with excess blasts-2	• Drug: AG-120 • Drug: Placebo or AG-120 • Drug: AG-221 • Drug: Placebo for AG-221	968	Phase 3
NCT03503409	IDH1 (AG 120) inhibitor in patients with IDH1-mutated myelodysplastic syndrome	• Myelodysplastic syndromes • Acute myeloid leukemia	• Drug: AG-120	68	Phase 2
NCT03471260	Ivosidenib and venetoclax with or without azacitidine in treating participants with IDH1-mutated hematologic malignancies	• High-risk myelodysplastic syndrome • IDH1.R132X • Myeloproliferative neoplasm • Recurrent/Refractory acute myeloid leukemia	• Drug: Azacitidine • Drug: Ivosidenib • Other: Pharmacokinetic Study • Drug: Venetoclax	48	Phase 1/2
NCT03245424	Ivosidenib expanded access program in relapsed/refractory aml with an IDH1 mutation	• Acute myeloid leukemia • Relapsed adult AML • Relapsed pediatric AML	• Drug: Ivosidenib (AG-120)
NCT03173248	Study of AG-120 (ivosidenib) vs placebo in combination with azacitidine in patients with previously untreated acute myeloid leukemia with an IDH1 mutation	• Newly diagnosed acute myeloid leukemia • Untreated AML • AML arising from myelodysplastic syndrome	• Drug: AG-120 (ivosidenib) with azacitidine • Drug: Placebo with azacitidine	200	Phase 3
NCT02677922	A safety and efficacy study of oral AG-120 plus subcutaneous azacitidine and oral AG-221 plus subcutaneous azacitidine in subjects with newly diagnosed acute myeloid leukemia (AML)	• Leukemia, myeloid, acute	• Drug: AG-120 • Drug: Azacitidine • Drug: AG-221	131*	Phase 1/2
NCT02632708	Safety study of AG-120 or AG-221 in combination with induction and consolidation therapy in participants with newly diagnosed acute myeloid leukemia (AML) with an IDH1 and/or IDH2 mutation	• Newly diagnosed acute myeloid leukemia • Untreated AML • AML arising from myelodysplastic syndrome (MDS) • AML arising from antecedent hematologic disorder • AML arising after exposure to genotoxic injury	• Drug: AG-120 • Drug: AG-221 • Drug: Cytarabine • Drug: Daunorubicin • Drug: Idarubicin • Drug: Mitoxantrone • Drug: Etoposide	153*	Phase 1
NCT02074839	Study of orally administered AG-120 in subjects with advanced hematologic malignancies with an IDH1 mutation	• Relapsed/Refractory acute myeloid leukemia • Untreated AML • Other IDH1-mutated Positive Hematologic Malignancies • Myelodysplastic Syndromes	• Drug: AG-120	291	Phase 1

Notes: Data from www.clinicaltrials.gov (January 2021). *Actual enrollment.

Figure 1 How to incorporate ivosidenib in AML treatment.