A phase 3, randomised, double-blind, placebo‑controlled international study in 717 patients (18 to <60 years of age) with newly diagnosed FLT3+ AML evaluated in 3 phases of treatment1,2
STEM CELL TRANSPLANTATION:
Sustained complete response provides appropriate patients the opportunity to proceed to stem cell transplant (SCT) during any phase of treatment
The study compared RYDAPT plus standard chemotherapy vs standard chemotherapy plus placebo, followed by single-agent maintenance therapy with RYDAPT vs placebo for 1 year (twelve 28-day cycles)1
The primary endpoint in RATIFY was overall survival (OS), measured from the date of randomisation until death by any cause1
The key secondary endpoint was event-free survival (EFS). Other secondary endpoints included: OS censored at SCT; SCT rate; complete remission (CR) rate; disease-free survival (DFS); and toxicity of the RYDAPT arm1,2
RYDAPT/placebo were administered in RATIFY throughout the 3 phases (induction, consolidation, and maintenance) of AML treatment1,2
Transplantation was allowed but not specifically mandated per study protocol. Patients who proceeded to haematopoietic SCT stopped receiving study treatment on or before the time of stem cell infusion, but all patients were followed for survival.
The study compared RYDAPT plus standard chemotherapy vs standard chemotherapy plus placebo, followed by single-agent maintenance therapy with RYDAPT vs placebo for 1 year (twelve 28-day cycles)1
The primary endpoint in RATIFY was overall survival (OS), measured from the date of randomisation until death by any cause1
The key secondary endpoint was event-free survival (EFS). Other secondary endpoints included: OS censored at SCT; SCT rate; complete remission (CR) rate; disease-free survival (DFS); and toxicity of the RYDAPT arm1,2
RYDAPT/placebo were administered in RATIFY throughout the 3 phases (induction, consolidation, and maintenance) of AML treatment1,2
Transplantation was allowed but not specifically mandated per study protocol. Patients who proceeded to haematopoietic SCT stopped receiving study treatment on or before the time of stem cell infusion, but all patients were followed for survival.
FLT3 mutational status (a stratification factor) was balanced between the two treatment arms: ITD allelic ratio <0.7 (46%), ITD allelic ratio ≥0.7 (31%), TKD (23%)2
Patients aged 18 to <60 years from 225 sites in 17 countries participated in RATIFY, spanning Europe, Australia, and North America, with two-thirds of randomised subjects from Europe and Australia2,3
Patients received SCT at a similar rate with RYDAPT plus standard chemotherapy compared with standard chemotherapy plus placebo (59.4% vs 55.2%)1
References: 1. RYDAPT [Summary of Product Characteristics]. Novartis Pharma AG; 2017. 2. Data on file. Study no. CPKC412A2301. Novartis Pharmaceuticals Corp; 2016. 3. Novartis Media Release. Novartis drug PKC412 (midostaurin) improves overall survival by 23% in global Phase III study of AML patients with FLT3 mutations. https://www.novartis.com/news/media-releases/novartis-drug-pkc412-midostaurin-improves-overall-survival-23-global-phase-iii. Published December 6, 2015. Accessed September 7, 2017.