NCT03826992 · Children's Hospital Medical Center, Cincinnati
Venetoclax Combined With Vyxeos (CPX-351) for Participants With Relapsed or Refractory Acute Leukemia
What this study is about
This study evaluates the safety and how well patients handle the treatment 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.
View original scientific description
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.
Interventions
DRUG
Vyxeos
Vyxeos Dose: daunorubicin 44 mg/m2 and cytarabine 100 mg/m2 administered via intravenous infusion over 90 minutes on Days 1, 3, and 5.
DRUG
Venetoclax
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
Primary outcome measures
Feasibility of combining venetoclax and Vyxeos (dose limiting toxicities)
Time frame: 28 days
If 2 or more participants have dose limiting toxicities at a given dose level, the maximum tolerated dose will have been exceeded.
Treatment related toxicities
Time frame: 60 days
Number of related adverse events
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Ages 1 Year to 39 Years
- Diagnosis of one of the following:
- Acute myeloid leukemia (AML), any subtype except
- Patients with acute promyelocytic leukemia (APML) are NOT eligible
- Patients with ML-DS are NOT eligible
- Myeloid sarcoma
- Acute leukemia of ambiguous lineage (ALAL)
- Acute undifferentiated leukemia (AUL)
- T/myeloid mixed phenotype acute leukemia (MPAL)
- B/myeloid MPAL
- MPAL with KMT2A-rearrangement MPAL with t (9;22) are NOT eligible
- T-cell acute lymphoblastic leukemia (T ALL)
- Early thymocyte precursor (ETP) ALL
- KMT2A-rearranged ALL
- Disease Status
- Relapsed/Refractory AML, MPA, and AUL
- Untreated therapy related AML
- Relapsed/Refractory KMT2A-rearranged ALL, T-cell ALL, ETEP ALL
- Karnofsky/Lanksy performance level score of greater than or equal to 50 percent.
- Prior therapy requirements
- Fully recovered from acute toxicities of Hematopoietic Stem Cell Transplant (HSCT) or Anthracycline Exposure
- 14 days must have elapsed since the completion of systemic cytotoxic therapy other than hydroxyurea, decitabine or azacitidine
- 2 weeks must have elapsed for local palliative radiotherapy (RT); 6 months must have elapsed if prior craniospinal RT or if 50% radiation of pelvis, and at least 6 weeks must have elapsed if other substantial bone marrow radiation
- Adequate renal, liver, cardiac, and central nervous system (CNS) function
Exclusion criteria
- Diagnosis of one of the following:
- Myeloid Leukemia associated with Down Syndrome (ML-DS)
- Acute Promyelocytic Leukemia (APML)
- Acute leukemia with CNS status 3 involvement
- Philadelphia chromosome t(9;22) positive leukemia (Ph+ ALL, AML, MPAL, or AUL)
- Fanconi Anemia, Shwachman-Diamond syndrome, or any other bone marrow failure syndrome or DNA repair disorder
- Wilson's Disease or other copper-metabolism disorder
- Pregnant or breastfeeding
- Uncontrolled infection
- Received greater than 13.6 Gray (Gy) prior radiation to the mediastinum
- Receipt of growth factors within 7 days prior to enrollment
- Currently receiving another investigational drug
- Currently receiving anti-cancer agents (with the exception of intrathecal (IT) agents or hydroxyurea)
- Unable to comply with the safety monitoring requirements of the study
Where
- Cincinnati, Ohio
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Feb 18, 2026 · Source of record for eligibility and locations