Study of Chemotherapy in Combination With All-trans Retinoic Acid (ATRA) With or Without Gemtuzumab Ozogamicin in Patients With Acute Myeloid Leukemia (AML) and Mutant Nucleophosmin-1 (NPM1) Gene Mutation
Summary
Randomized Phase-III, two-arm, open-label, multi-center study in adult patients with AML and NPM1 mutation. Before Amendment No. 4 (December 2013): Primary Efficacy Objective: * Evaluation of efficacy based on event-free survival (EFS) after induction and consolidation chemotherapy plus all-trans retinoic acid (ATRA) with or without gemtuzumab ozogamicin (GO) in adult patients with acute myeloid leukemia (AML) and mutant nucleophosmin-1 (NPM1) After Amendment No. 4 (December 2013): Primary Efficacy Objective: * Evaluation of efficacy based on overall survival (OS) after induction and consolidation chemotherapy plus all-trans retinoic acid (ATRA) with or without gemtuzumab ozogamicin (GO) in adult patients with acute myeloid leukemia (AML) and mutant nucleophosmin-1 (NPM1)
Timeline
- Start
- 2010-05-12
- Primary completion
- 2021-09-01
- Completion
- 2021-09-01
Publications
- Miah K, Goeman JJ, Putter H, Kopp-Schneider A, Benner A. Variable Selection via Fused Sparse-Group Lasso Penalized Multi-state Models Incorporating Molecular Data. Biom J. 2025 Dec;67(6):e70087. doi: 10.1002/bimj.70087.
- Dohner H, Weber D, Krzykalla J, Fiedler W, Kuhn MWM, Schroeder T, Mayer K, Lubbert M, Wattad M, Gotze K, Fransecky L, Koller E, Wulf G, Schleicher J, Ringhoffer M, Greil R, Hertenstein B, Krauter J, Martens UM, Nachbaur D, Samra MA, Machherndl-Spandl S, Basara N, Leis C, Schrade A, Kapp-Schwoerer S, Cocciardi S, Bullinger L, Thol F, Heuser M, Paschka P, Gaidzik VI, Saadati M, Benner A, Schlenk RF, Dohner K, Ganser A; German-Austrian AML Study Group. Intensive chemotherapy with or without gemtuzumab ozogamicin in patients with NPM1-mutated acute myeloid leukaemia (AMLSG 09-09): a randomised, open-label, multicentre, phase 3 trial. Lancet Haematol. 2023 Jul;10(7):e495-e509. doi: 10.1016/S2352-3026(23)00089-3. Epub 2023 May 12.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Gemtuzumab Ozogamicin | Monoclonal antibody | 3 mg/m2 | Intravenous |
| Background | Cytarabine | Small molecule | — | — |
| Background | Etoposide | Small molecule | — | — |
| Background | Idarubicin | Small molecule | — | — |
| Background | Pegfilgrastim | Protein / enzyme biologic | — | — |
| Background | Tretinoin | Small molecule | — | — |