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Vyxeos

Phase 2

Acute Myeloid Leukaemia | Small molecule | Hematology |Jazz Pharmaceuticals plc|Last Updated: May 5, 2026

Success Probability

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Trial Design

RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment333

FDA Designations

No designations recorded

Clinical trial landscape

Vyxeos · 1 trial · 2 indications

Phase 2 1
NCT04217278A Trial of Treatments to Assess the Effects on Outcome of Adults With AML and MDS Undergoing Allogeneic SCTAcute Myeloid Leukaemia
ACTIVE NOT_RECRUITING333 Analytics
PHASE2ACTIVE NOT_RECRUITING
A Trial of Treatments to Assess the Effects on Outcome of Adults With AML and MDS Undergoing Allogeneic SCT
Acute Myeloid LeukaemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Overall survival (all randomisations)
12 and 24 months

Defined as time from entering the relevant randomisation to the relevant question until death from any cause. Patients who are alive at the end of the trial or have been lost to follow up will be censored at their date last seen. For randomisations 2 and 3 this outcome will also be calculated as time from transplantation in order to run a sensitivity analysis.

Secondary Endpoints

Change in MRD status - R1 only
Assessed at baseline and pre-transplant
Disease-free survival
From date of randomisation through to study completion, an average of 6 years
Cumulative incidence of disease relapse
From date of randomisation through to study completion, an average of 6 years
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
R1: Intermediate dose CytarabineACTIVE_COMPARATORFirst Randomisation (closed to recruitment) - control arm: Intermediate dose Cytarabine (1g/m\^2 administered by intravenous infusion over 2 hours on days 1-5 inclusive)
R1: VyxeosEXPERIMENTALFirst Randomisation (closed to recruitment) - experimental arm: Vyxeos (29mg/65mg/m\^2 administered by intravenous infusion over 90 minutes on days 1 and 3)
R2: FB4ACTIVE_COMPARATORSecond Randomisation - under 55 years - control arm: Fludarabine (40mg/m\^2 days -7, -6, -5, and -4), Busulphan (3.2mg/kg days -7, -6, -5 and -4)
R2: TBFEXPERIMENTALSecond Randomisation - under 55 years - experimental arm: Thiotepa (5mg/kg day -7 and -6), Busulphan (3.2mg/kg days -5, -4 and -3), Fludarabine (50mg/m\^2 days -5, -4 and -3)
R3: FB2ACTIVE_COMPARATORThird Randomisation - 55 years and over (or under 55 with comorbidities) - control arm: Fludarabine (30mg/m\^2 days -6, -5, -4, -3 and -2), Busulphan (3.2mg/kg days -6 and -5)
Mini-TBFEXPERIMENTALThird Randomisation - 55 years and over (or under 55 with comorbidities) - experimental arm: Thiotepa (5mg/kg day -6), Busulphan (3.2mg/kg days -5 and -4), Fludarabine (50mg/m\^2 days -5, -4, and -3)

Interventions

NameTypeDescription
VyxeosDRUGVyxeos administered by intravenous infusion
FludarabineDRUGFludarabine administered by intravenous infusion
BusulphanDRUGBusulphan administered by intravenous infusion
ThiotepaDRUGThiotepa administered by intravenous infusion
CytarabineDRUGCytarabine administered by intravenous infusion
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites14

Eligibility Criteria for Randomisation 1 (closed to recruitment) Inclusion Criteria for Randomisation 1 1. Patients (≥ 18 years old) with a morphological documented diagnosis of AML or MDS who are deemed fit for allo-SCT with one of the following disease characteristics: AML * Patients in 1s...

Countries:United Kingdom
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Frequently asked questions about Vyxeos

What is Vyxeos used for?

Vyxeos is a liposome injection being studied for use in acute myeloid leukemia (AML) and relapsed or refractory pediatric AML. It is also being evaluated in high-risk myelodysplastic syndrome in the context of allogeneic stem cell transplantation. Vyxeos is an investigational small molecule therapy in clinical development.

Who makes Vyxeos?

Vyxeos is developed by Jazz Pharmaceuticals plc, a biopharmaceutical company traded on the NASDAQ under the ticker JAZZ. The company is conducting clinical trials to evaluate Vyxeos in hematologic malignancies, including acute myeloid leukemia and relapsed pediatric AML.

What phase is Vyxeos in?

Vyxeos is in Phase 2 clinical development for acute myeloid leukemia and high-risk myelodysplastic syndrome. A separate Phase 1 trial is evaluating Vyxeos in combination with clofarabine for relapsed or refractory pediatric AML. Vyxeos remains investigational and is not yet approved by regulatory authorities.

What clinical trials is Vyxeos in?

Vyxeos is being studied in two clinical trials. NCT04217278 is a Phase 2 trial in adults with AML and high-risk myelodysplastic syndrome undergoing allogeneic stem cell transplantation, enrolling 333 participants in the United Kingdom. NCT07156435 is a Phase 1 trial combining Vyxeos with clofarabine in pediatric patients with relapsed or refractory AML across multiple European countries.

Is Vyxeos the same as Vyxeos 44 MG / 100 MG Liposome Injection?

Yes, Vyxeos is also known as Vyxeos 44 MG / 100 MG Liposome Injection and Vyxeos/ Liposome. These names refer to the same liposomal formulation of the drug. The 44 mg and 100 mg designations indicate the two active components in the liposome injection.

What does Vyxeos target?

Vyxeos is a liposome injection containing two chemotherapy agents, daunorubicin and cytarabine, in a synergistic 1:5 molar ratio. The liposomal formulation is designed to deliver both drugs to leukemia cells. Vyxeos is being studied in acute myeloid leukemia and related hematologic conditions.