Table 2.

Selected ongoing clinical trials of midostaurin

Name	Phase	Patient population	Treatment	Comments
NCT00651261	III	AML (FLT3-mutated) newly diagnosed age <60 years	Induction and consolidation chemotherapy ± midostaurin	First Phase III trial of midostaurin
NCT01883362	II	AML (FLT3-mutated) post-HCT	HCT followed by standard of care ± midostaurin 50 mg PO BID ×12 months	First trial of midostaurin maintenance after HCT
NCT01477606	II	AML (FLT3-mutated)	Midostaurin added to standard induction and consolidation therapy (including HCT) + maintenance midostaurin	First trial of midostaurin during induction, consolidation, and maintenance treatment of AML
NCT00233454	II	Aggressive systemic mastocytosis or mast cell leukemia	Midostaurin 100 mg PO BID	First trials of midostaurin in mastocytosis
NCT00782067	II	Aggressive systemic mastocytosis or mast cell leukemia ± an associated hematologic clonal non–mast cell lineage disease	Midostaurin 100 mg PO BID
NCT01920204	II	Indolent or smoldering systemic mastocytosis with D816V mutation	Midostaurin 100 mg PO BID ×6 months
NCT00866281	I/II	AML with FLT3 mutation (relapsed/refractory) or ALL with MLL rearrangement (relapsed/refractory)	Midostaurin PO BID (dose escalation)	First pediatric trial of midostaurin
NCT01429337	I	Healthy volunteers with normal or impaired hepatic function	Midostaurin 25 mg PO BID days 1–6, 25 mg PO daily on day 7	First trial of midostaurin in patients with hepatic impairment Patients not required to have an underlying hematologic malignancy

Abbreviations: AML, acute myeloid leukemia; ALL, acute lymphoblastic leukemia; PO, by mouth; BID, twice daily; HCT, hematopoietic cell transplant; FLT3, fms-like tyrosine kinase; MLL, mixed lineage leukemia.