drugset / Trial / NCT00782067
Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia
NaSingle-groupOpen-labelTreatment
Summary
The purpose of this study was to determine the efficacy and safety of twice daily (bid) oral midostaurin in patients with Aggressive Systemic Mastocytosis (ASM) or Mast Cell Leukemia (MCL) with or without an Associated Hematological clonal Non-Mast cell lineage Disease (AHNMD).
Timeline
- Start
- 2008-10-13
- Primary completion
- 2014-12-01
- Completion
- 2017-08-24
Outcome
Met primary endpoint
registry NUMBER Midostaurin (PKC412): 59.6 (48.6 to 69.8) Percentage of participants NCT00782067 ↗
registry analysis (other test); Midostaurin (PKC412); p <0.001; Exact Binomial Test; note: Null hypothesis: ORR \<= 30% Alternative hypothesis: ORR \>= 50% NCT00782067 ↗
Publications
- Medawar G, Sakalabaktula K, Magri J, Rinker E, Baratam P. KIT V560D-Mutated Systemic Mastocytosis Associated With High-Risk Myelodysplastic Syndrome: A Unique Case of Systemic Mastocytosis-Associated Hematologic Neoplasm. Case Rep Hematol. 2024 Nov 30;2024:4360304. doi: 10.1155/crh/4360304. eCollection 2024.
- Hartmann K, Gotlib J, Akin C, Hermine O, Awan FT, Hexner E, Mauro MJ, Menssen HD, Redhu S, Knoll S, Sotlar K, George TI, Horny HP, Valent P, Reiter A, Kluin-Nelemans HC. Midostaurin improves quality of life and mediator-related symptoms in advanced systemic mastocytosis. J Allergy Clin Immunol. 2020 Aug;146(2):356-366.e4. doi: 10.1016/j.jaci.2020.03.044. Epub 2020 May 11.
- Gotlib J, Kluin-Nelemans HC, George TI, Akin C, Sotlar K, Hermine O, Awan FT, Hexner E, Mauro MJ, Sternberg DW, Villeneuve M, Huntsman Labed A, Stanek EJ, Hartmann K, Horny HP, Valent P, Reiter A. Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis. N Engl J Med. 2016 Jun 30;374(26):2530-41. doi: 10.1056/NEJMoa1513098.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Midostaurin | Small molecule | 100 mg | Oral |