A Phase 1 interventional study of Vyxeos and Venetoclax in Leukemia, sponsored by Children's Hospital Medical Center, Cincinnati. Recruiting at 1 site in United States. Open to participants aged 1 Year to 39 Years. Per ClinicalTrials.gov, last updated 2026-02-18.
Sponsored by Children's Hospital Medical Center, Cincinnati · Phase 1, Interventional, and Treatment
This study evaluates the safety and tolerability of combining venetoclax with Vyxeos (CPX-351) in pediatric and young adult patients with acute leukemia that has come back or not responded to treatment.
This is a single-institution Phase I pilot study designed to test the safety and tolerability of combining venetoclax with Vyxeos (CPX-351, cytarabine and daunorubicin liposome) for the treatment of relapsed/refractory acute leukemia in young patients. Subjects will receive a single course of study therapy consisting of daily, oral or crushed venetoclax at an assigned dose level with a 3-day ramp-up to target dose and Vyxeos administered intravenously at the established dose on Days 1, 3, and 5. In addition to safety and tolerability, the overall response rate to these therapies will be estimated. Pharmacokinetic (PK) analysis will also be conducted to define the drug clearance of venetoclax in this combination.
5,442 studies on the registry are indexed under Leukemia; 637 are open to participants now.
This study's planned enrollment of 21 is below the median of 38 across 4,248 interventional studies indexed under Leukemia.
Browse Leukemia studies →Children's Hospital Medical Center, Cincinnati is the lead sponsor of 661 studies on the registry; 134 are open to participants now.
Of its 54 completed or terminated interventional studies of FDA-regulated products, 30 (56%) have results posted.
Counted across the registry records on this site, refreshed daily.
Diagnosis of one of the following:
Acute myeloid leukemia (AML), any subtype except
Acute leukemia of ambiguous lineage (ALAL)
Disease Status
Prior therapy requirements
Exclusion Criteria:
Diagnosis of one of the following:
Venetoclax will be given orally on Days per the assigned dose level. A single course consisting of 3 doses of Vyxeos and 7-21 doses of venetoclax depending on the assigned dose level will be administered to participants in this study. Vyxeos will be administered by central venous catheter over 90 minutes on Day 1, 3, and 5. Venetoclax is given daily by mouth per assigned dose level.
Drug: Vyxeos · Drug: Venetoclax
Vyxeos Dose: daunorubicin 44 mg/m2 and cytarabine 100 mg/m2 administered via intravenous infusion over 90 minutes on Days 1, 3, and 5.
Also known as: cytarabine and daunorubicin liposome, CPX-351
Venetoclax Dose: 1. Dose Level 0 - weight based daily dosing for 21 days 2. Dose Level -1 - weight based daily dosing for 14 days 3. Dose Level -2- weight based daily dosing for 10 days 4. Dose Level -3- weight based daily dosing for 7 days
Also known as: Venclexta
Feasibility of combining venetoclax and Vyxeos (dose limiting toxicities)
If 2 or more participants have dose limiting toxicities at a given dose level, the maximum tolerated dose will have been exceeded.
Time frame: 28 days
Treatment related toxicities
Number of related adverse events
Time frame: 60 days
Disease response
Estimate of overall response rate (ORR) defined as (CR/CRi/CRp).
Time frame: 42 days
Cancer therapeutics-related cardiac dysfunction (CTRCD) in patients who have previously received anthracyclines
Measured by echocardiogram (ECHO)
Time frame: 60 days
Plan to share: No
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Children's Hospital Medical Center, Cincinnati