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Vyxeos/ Liposome

Phase 1

Relapsed Pediatric AML | Small molecule | Hematology |Jazz Pharmaceuticals plc|Last Updated: Sep 5, 2025

Target and mechanism

ModalitySmall molecule

Also known as Vyxeos

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment25

FDA Designations

No designations recorded

Clinical trial landscape

Vyxeos/ Liposome · 1 trial · 2 indications

Phase 1 1
NCT07156435Vyxeos® With Clofarabine for Pediatric AMLRelapsed Pediatric AML
RECRUITING25 Analytics
PHASE1RECRUITING
Vyxeos® With Clofarabine for Pediatric AML
Relapsed Pediatric AMLUnlock trial analytics

Study Endpoints

Primary Endpoints

Dose finding for combination Vyxeos® with clofarabine
2 years

To establish the recommended phase 2 dose of Vyxeos®/CPX-351 in combination with clofarabine in children with relapsed/refractory AML

Secondary Endpoints

To determine the safety and tolerability of Vyxeos®/CPX-351 in combination with clofarabine by observing dose-limiting toxicities (DLTs)
4 years
To determine the (preliminary) efficacy in terms of the hematological remission rate in these patients as determined by morphology with flow cytometric confirmation
4 years
To describe the durability of response, including the number of patients that undergo stem- cell transplant after re-induction with this regimen
4 years
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Treatment with Vyxeos®/CPX-351 in combination with clofarabineEXPERIMENTALTreatment will consist of 2 courses. An adapted regimen is used to combine Vyxeos®/CPX-351 at a fixed dose given at day 1, 3, 5 with clofarabine at the allocated dose level given at day 2-6 in course one, and only Vyxeos®/CPX-351 in course 2. A maximum of 3 dose levels of clofarabine are expected to be tested in this study.

Interventions

NameTypeDescription
Vyxeos 44 MG / 100 MG Liposome InjectionDRUGVyxeos®/CPX-351 will be infused in 90 minutes on day 1, 3 and 5 only, 3 hours after the end of clofarabine (if administered on the same day).
ClofarabineDRUGClofarabine infusion will be given according to the assigned dose level, over 2 hours IV, daily on day 2-6 (for 5 consecutive days).
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Eligibility Criteria

Age Range1 Year to 21 Years
SexALL
Healthy VolunteersNo
Study Sites13

We will include pediatric patients ≥1 year and ≤21 years with: * Any ≥ 2nd relapse of AML * Refractory AML (defined as ≥ 20% blasts in the bone marrow after standard (re-) induction therapy) * Early 1st relapse (defined as relapse within one year from initial diagnosis) of AML * Any relapse of AML ...

Countries:AustriaDenmarkGermanyItalyNetherlandsSpain
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Frequently asked questions about Vyxeos/ Liposome

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.