Vyxeos (cytarabine/daunorubicin liposomal formulation)

P3, N=1186, Recruiting, Children's Oncology Group | Trial completion date: Sep 2027 --> Jun 2029 | Trial primary completion date: Sep 2027 --> Jun 2029

P=N/A, N=113, Completed, Jazz Pharmaceuticals | Active, not recruiting --> Completed | Trial completion date: Feb 2026 --> Oct 2025 | Trial primary completion date: Feb 2026 --> Oct 2025

P2, N=46, Suspended, Roswell Park Cancer Institute | Recruiting --> Suspended

For patients entering the course-2 randomisation (n=107) survival was equivalent between standard versus intensified CPX doses (P=0.565).In this population of older patients without known adverse-risk cytogenetics, DAGO2 resulted in superior survival compared to CPX. CPX did not benefit those with MDS-related mutations over DAGO2.

P2, N=50, Active, not recruiting, M.D. Anderson Cancer Center | Trial completion date: Nov 2025 --> Nov 2027 | Trial primary completion date: Nov 2025 --> Nov 2027

Some therapies have already been developed for actionable mutations: quizartinib for FLT3-ITD mutations is available for newly diagnosed AML, and ivosidenib for IDH1 mutations is awaiting approval...Decisions on allogeneic transplantation and accessibility of investigational drugs are also expected. Detailed diagnosis and prognosis prediction based on the profile of genetic abnormalities should enable precision medicine.

The most common adverse events were related to myelosuppression. CPX + VEN resulted in high remission rates and enabled progression to allogenic SCT for the majority of a highly adverse group of ND AML patients.

Intensity-reduced induction and reinduction therapy with cytarabine, idarubicin ± etoposide of the ML-DS 2006 trial was replaced with CPX-351 (66 U/m² on three days in course 1 and two days in course 2)...In conclusion, replacing intensity-reduced induction therapy with CPX-351 in ML-DS resulted in significantly lower event-free survival, highlighting the need for dose optimization to balance efficacy and toxicity in this sensitive patient population. EudraCT: 2018-002988-25.

P2, N=25, Active, not recruiting, St. Jude Children's Research Hospital | Recruiting --> Active, not recruiting

We evaluated the safety and efficacy of the combination of daunorubicin, cytarabine (DA), gemtuzumab ozogamicin (GO) and midostaurin (DAGO+m) for younger patients with newly diagnosed FLT3mut AML in the UK NCRI AML19 trial...DAGO2+m will now be evaluated in a randomised study (OPTIMISE-FLT3, ISRCTN 34016918). Trial: ISRCTN78449203.

Our analysis suggests that also s-AML patients with NPM1 mutations and those belonging to the ELN 2017 favorable risk category benefit from CPX-351. In eligible patients, allo-HSCT should be performed as soon as a CR is achieved, whereas patients not undergoing transplant benefit from a complete CPX-351 schedule.

P1, N=22, Recruiting, H. Lee Moffitt Cancer Center and Research Institute | Trial primary completion date: Jan 2025 --> Feb 2027 | Trial completion date: May 2026 --> Mar 2027