Venetoclax Combined With Vyxeos (CPX-351) for Participants With Relapsed or Refractory Acute Leukemia
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ClinicalTrials.gov Identifier: NCT03826992 |
Recruitment Status :
Suspended
(Interim Analysis)
First Posted : February 1, 2019
Last Update Posted : January 14, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Leukemia | Drug: Vyxeos Drug: Venetoclax | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 18 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase I Study of Venetoclax Combined With Vyxeos (CPX-351) for Children, Adolescents and Young Adults With Relapsed or Refractory Acute Leukemia |
Actual Study Start Date : | December 27, 2018 |
Estimated Primary Completion Date : | January 2023 |
Estimated Study Completion Date : | January 2024 |

Arm | Intervention/treatment |
---|---|
Experimental: Venetoclax and Vyxeos combination
Venetoclax will be given orally on Days 1-21 per the assigned dose level. A single course consisting of 3 doses of Vyxeos and 21 doses of venetoclax 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
Vyxeos Dose: daunorubicin 44 mg/m2 and cytarabine 100 mg/m2 administered via intravenous infusion over 90 minutes on Days 1, 3, and 5.
Other Name: cytarabine and daunorubicin liposome, CPX-351 Drug: Venetoclax Venetoclax Dose:
Other Name: Venclexta |
- 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
- Disease response [ Time Frame: 42 days ]Estimate of overall response rate (ORR) defined as (CR/CRi/CRp).
- Cancer therapeutics-related cardiac dysfunction (CTRCD) in patients who have previously received anthracyclines [ Time Frame: 60 days ]Measured by echocardiogram (ECHO)

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Ages Eligible for Study: | 1 Year to 39 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Ages 1-39 years
-
Diagnosis of one of the following:
- Acute myeloid leukemia (AML)
- Acute undifferentiated leukemia (AUL)
- Mixed phenotype acute leukemia (MPAL)
- T-cell acute lymphoblastic leukemia (T ALL)
- Early thymocyte precursor (ETP) ALL
- KMT2A-rearranged ALL
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Disease status
- Relapsed/Refractory AML, MPAL and AUL
- Relapsed/Refractory KMT2A-rearranged ALL, T-cell ALL, ETP ALL
- Karnofsky/ Lanksy performance level score of greater than or equal to 50 percent
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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:
- Acute Promyelocytic Leukemia (APML)
- Acute leukemia with CNS status 3 involvement
- Philadelphia chromosome positive leukemia (Ph+ ALL, 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
- Unable to swallow tablets
- 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03826992
United States, Ohio | |
Cincinnati Children's Hospital Medical Center | |
Cincinnati, Ohio, United States, 45229 |
Principal Investigator: | John Perentesis, MD | Children's Hospital Medical Center, Cincinnati |
Responsible Party: | Children's Hospital Medical Center, Cincinnati |
ClinicalTrials.gov Identifier: | NCT03826992 |
Other Study ID Numbers: |
V2-MA-1801 |
First Posted: | February 1, 2019 Key Record Dates |
Last Update Posted: | January 14, 2022 |
Last Verified: | December 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
relapsed refractory Vyxeos Venetoclax acute myeloid leukemia acute myeloid leukemia, childhood |
mixed-lineage leukemia (MLL) AML CPX-351 Venclexta Histone-lysine N-methyltransferase 2A (KmT2A) |
Leukemia Neoplasms by Histologic Type Neoplasms Cytarabine Daunorubicin Venetoclax Antimetabolites, Antineoplastic Antimetabolites Molecular Mechanisms of Pharmacological Action Antineoplastic Agents |
Antiviral Agents Anti-Infective Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Antibiotics, Antineoplastic Topoisomerase II Inhibitors Topoisomerase Inhibitors Enzyme Inhibitors |