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Vyxeos / Liposome

Phase 1

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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment25
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07156435Vyxeos® With Clofarabine for Pediatric AMLPHASE1 RECRUITING 25Nov 6, 2020Dec 31, 2028Sep 5, 202513 Austria, Denmark +4
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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 — 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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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT07156435studyFirstPostDate: changed