Numerous clinical trials spanning Phase 1 through Phase 3 evaluate midostaurin across hematological indications, primarily acute myeloid leukemia, myelodysplastic syndromes, and FLT3-mutated leukemias. Sponsored by pharmaceutical companies such as Novartis Pharmaceuticals, academic institutions, and cooperative research groups, these studies reflect recruitment statuses ranging from active and recruiting to completed, terminated, and active not recruiting. Ongoing research continues to assess therapeutic combinations and resource utilization within these patient populations.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT04982354 | WITHDRAWN | Acute Myeloid Leukemia |
Guenther Koehne |
2022-07-05 | PHASE1; PHASE2 |
| NCT02115295 | RECRUITING | Acute Biphenotypic Leukemia; Acute Myeloid Leukemia; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Blasts 10 Percent or More of Bone Marrow Nucleated Cells; Blasts 10 Percent or More of Peripheral Blood White Cells; de Novo Myelodysplastic Syndrome; Myelodysplastic Syndrome; Previously Treated Myelodysplastic Syndrome; Recurrent Acute Myeloid Leukemia; Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Refractory Acute Myeloid Leukemia; Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Secondary Acute Myeloid Leukemia; Untreated Adult Acute Myeloid Leukemia |
M.D. Anderson Cancer Center |
2014-05-19 | PHASE2 |
| NCT03591510 | ACTIVE_NOT_RECRUITING | FLT3-mutated Acute Myeloid Leukemia |
Novartis Pharmaceuticals |
2019-03-13 | PHASE2 |
| NCT07726576 | NOT_YET_RECRUITING | Leukemia, Myeloid, Acute |
University Hospital, Bordeaux |
2026-09 | PHASE1; PHASE2 |
| NCT04385290 | RECRUITING | Acute Myeloid Leukemia |
Technische Universität Dresden |
2020-09-04 | PHASE1; PHASE2 |
| NCT06313437 | RECRUITING | Acute Myeloid Leukemia; AML, Adult; AML With Gene Mutations; AML; Leukemia |
Richard Stone, MD |
2024-12-06 | PHASE1 |
| NCT03258931 | COMPLETED | Newly Diagnosed FLT3 Mutated AML |
Arog Pharmaceuticals, Inc. |
2018-08-15 | PHASE3 |
| NCT04027309 | ACTIVE_NOT_RECRUITING | Acute Myeloid Leukemia; Myelodysplastic Syndrome With Excess Blasts-2 |
Stichting Hemato-Oncologie voor Volwassenen Nederland |
2019-12-20 | PHASE3 |
| NCT03900949 | COMPLETED | Acute Myeloid Leukemia |
Uma Borate |
2019-03-13 | PHASE1 |
| NCT03836209 | COMPLETED | Acute Myeloid Leukemia |
PrECOG, LLC. |
2019-12-06 | PHASE2 |
| NCT03092674 | COMPLETED | Acute Myeloid Leukemia; Myelodysplastic Syndrome; Myelodysplastic Syndrome/Acute Myeloid Leukemia |
National Cancer Institute (NCI) |
2018-02-02 | PHASE2; PHASE3 |
| NCT03686345 | TERMINATED | Core Binding Factor Acute Myeloid Leukemia (CBF-AML) |
Niguarda Hospital |
2018-07-01 | PHASE2 |
| NCT04174612 | UNKNOWN | Acute Myeloid Leukemia With FLT3/ITD Mutation |
Gruppo Italiano Malattie EMatologiche dell'Adulto |
2020-04-24 | PHASE3 |
| NCT03280030 | COMPLETED | Acute Myeloid Leukemia |
Novartis Pharmaceuticals |
2018-04-06 | PHASE2 |
| NCT04496999 | TERMINATED | AML, Adult |
Insel Gruppe AG, University Hospital Bern |
2020-07-13 | PHASE1 |
| NCT04075747 | COMPLETED | Acute Myeloid Leukemia |
Jazz Pharmaceuticals |
2019-12-02 | PHASE1 |
| NCT04097470 | ACTIVE_NOT_RECRUITING | AML/MDS |
Stichting Hemato-Oncologie voor Volwassenen Nederland |
2019-12-05 | PHASE2 |
| NCT03760445 | WITHDRAWN | Leukemia, Myeloid, Acute |
Novartis Pharmaceuticals |
2019-11-15 | PHASE1; PHASE2 |
| NCT03379727 | COMPLETED | Acute Myeloid Leukemia |
Novartis Pharmaceuticals |
2018-02-13 | PHASE3 |
| NCT03951961 | TERMINATED | Acute Myeloid Leukemia, Adult |
University of Jena |
2020-03-20 | PHASE2 |
| NCT03512197 | COMPLETED | Acute Myeloid Leukemia (AML) |
Novartis Pharmaceuticals |
2018-07-20 | PHASE3 |
| NCT03207334 | WITHDRAWN | Acute Myeloid Leukemia |
University of Florida |
2018-11 | PHASE2 |
| NCT05488613 | Completed | Acute Myeloid Leukemia (AML) |
Novartis Pharmaceuticals|Novartis |
2020-09-24 | -- |
| NCT01429337 | COMPLETED | Hepatic Impairment |
Novartis Pharmaceuticals |
2011-03-07 | PHASE1 |
| NCT03951961 | Terminated | Acute Myeloid Leukemia Adult |
University of Jena|Ludwig-Maximilians - University of Munich |
2020-03-20 | Phase 2 |
| NCT01477606 | COMPLETED | Acute Myeloid Leukemia |
University of Ulm |
2012-05 | PHASE2 |
| NCT04097470 | Active not recruiting | AML/MDS |
Stichting Hemato-Oncologie voor Volwassenen Nederland|Swiss Group for Clinical Cancer Research |
2019-12-05 | Phase 2 |
| NCT01830361 | COMPLETED | Acute Myeloid Leukemia |
Technische Universität Dresden |
2013-03-13 | PHASE2 |
| NCT02078609 | COMPLETED | AML and High Risk MDS |
Novartis Pharmaceuticals |
2014-03-20 | PHASE1 |
| NCT01883362 | COMPLETED | Acute Myeloid Leukemia |
Novartis Pharmaceuticals |
2014-02-06 | PHASE2 |
| NCT02723435 | WITHDRAWN | Acute Myeloid Leukemia With Gene Mutations; Adult Acute Myeloid Leukemia in Remission |
Stanford University |
PHASE2 | |
| NCT02634827 | TERMINATED | Acute Myeloid Leukemia With FLT3/ITD Mutation; Acute Myeloid Leukemia With Gene Mutations; FLT3 Tyrosine Kinase Domain Point Mutation; Secondary Acute Myeloid Leukemia; Untreated Adult Acute Myeloid Leukemia |
Mayo Clinic |
2015-12-30 | PHASE2 |
| NCT01093573 | COMPLETED | Untreated Adult Acute Myeloid Leukemia |
Brenda Cooper, MD |
2009-07 | PHASE1; PHASE2 |
| NCT01846624 | TERMINATED | Acute Myeloid Leukemia (AML) With Multilineage Dysplasia Following Myelodysplastic Syndrome, in Adults; AML (Adult) With 11q23 (MLL) Abnormalities; AML (Adult) With Del (5q); AML (Adult) With Inv (16) (p13; q22); AML (Adult) With t (16;16) (p13; q22); AML (Adult) With t (8; 21) (q22; q22); Secondary AML (Adult); Untreated AML (Adult) |
David Iberri |
2013-06 | PHASE2 |
| NCT00651261 | UNKNOWN | Leukemia |
Alliance for Clinical Trials in Oncology |
2008-04 | PHASE3 |
| NCT01920204 | UNKNOWN | Indolent Systemic Mastocytosis |
University Medical Center Groningen |
2013-08 | PHASE2 |
| NCT01282502 | COMPLETED | Adenocarcinoma of the Rectum |
Massachusetts General Hospital |
2011-08 | PHASE1 |
| NCT00782067 | COMPLETED | Leukemia |
Novartis Pharmaceuticals |
2008-10-13 | PHASE2 |
| NCT00866281 | TERMINATED | Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia |
Novartis Pharmaceuticals |
2009-09 | PHASE1; PHASE2 |
| NCT01174888 | COMPLETED | Acute Myeloid Leukemia; Acute Myeloid Leukemia With Multilineage Dysplasia Following; Myelodysplastic Syndrome; Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities; Adult Acute Myeloid Leukemia With Del(5q); Adult Acute Myeloid Leukemia With Inv(16)(p13;q22); Adult Acute Myeloid Leukemia With t(15;17)(q22;q12); Adult Acute Myeloid Leukemia With t(16;16)(p13;q22); Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); Recurrent Adult Acute Myeloid Leukemia; Secondary Acute Myeloid Leukemia |
Alison Walker |
2010-08 | PHASE1 |
| NCT01130662 | COMPLETED | Acute Myeloid Leukemia |
University of Kansas |
2010-03 | PHASE1 |
| NCT01161550 | COMPLETED | Leukemia, Myeloid, Acute |
Washington University School of Medicine |
2010-11 | PHASE1 |
| NCT00093600 | COMPLETED | Acute Myeloid Leukemia (AML) |
Novartis Pharmaceuticals |
2004-02 | PHASE1 |
| NCT00233454 | COMPLETED | Systemic Mastocytosis, Aggressive (ASM); Leukemia, Mast Cell; Hematological Non-mast Cell Lineage Disease (AHNMD) |
Jason Robert Gotlib |
2005-03 | PHASE2 |
(data from https://clinicaltrials.gov, updated on 2024-10-01)
Check the
Midostaurin (PKC412)
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Mechanism and Biochemical Profile
Appendix RUO and cGMP Quality Standards
| Quality Dimension | RUO (Research Use Only) | cGMP (Current Good Manufacturing Practice) |
|---|---|---|
| Clinical Applicability | Prohibited in human clinical trials or medical diagnostics. | Mandatory for human clinical trials (Phase I–III) and therapies. |
| Regulatory Status | Non-regulated grade; exempt from drug manufacturing laws. | Legally enforced by health authorities (e.g., FDA, EMA, NMPA). |
| Facility Environment | Unclassified analytical or research laboratories. | Validated Cleanrooms (ISO Class 5–8) with continuous monitoring. |
| Quality Control | Basic purity and activity testing. | Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma). |
| Process Validation | Basic equipment calibration; no process validation required. | Full qualification (IQ/OQ/PQ) and complete batch records. |
| Quality Assurance | Vendor self-declared without required formal QMS. | Mandatory QA/QC unit, Change Control, CAPA, and vendor audits. |
| Regulatory Impact | High risk of IND rejection if used as a critical raw material. | Required for IND/NDA filings, supported by Drug Master Files (DMF). |
Footnotes
Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).
- Midostaurin (PKC412) Mechanism of Action: FLT3 Inhibition in Leukemic Blast Cells Research
- Midostaurin (PKC412) Solubility in DMSO
- Midostaurin (PKC412) Stock Solution
- Midostaurin (PKC412) Storage
- Midostaurin (PKC412) Stability
- Midostaurin (PKC412) Molecular Weight
- Midostaurin (PKC412) SMILES
- Midostaurin (PKC412) CAS Number
- Midostaurin (PKC412) Chemical Structure (2D and 3D)
- Midostaurin (PKC412) Sigma (569397)? Try a better option.
- Midostaurin (PKC412) MedChemExpress (HY-10230)? Try a better option.
- Midostaurin (PKC412) Cayman (10459)? Try a better option.
- Midostaurin (PKC412) APExBIO (B3709)? Try a better option.
- Midostaurin (PKC412) SDS｜MSDS
Note: Technical data last updated: Sep 1, 2026.