Venetoclax Combined With Vyxeos (CPX-351) for Participants With Relapsed or Refractory Acute Leukemia

February 16, 2026 updated by: Children's Hospital Medical Center, Cincinnati

A Phase I Study of Venetoclax Combined With Vyxeos (CPX-351) for Children, Adolescents and Young Adults With Relapsed or Refractory Acute Leukemia

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Estimated)

21

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital Medical Center
        • Principal Investigator:
          • John Perentesis, MD
        • Sub-Investigator:
          • Laura Agresta, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 35 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Venetoclax and Vyxeos combination

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
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 Names:
  • cytarabine and daunorubicin liposome, CPX-351
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
Other Names:
  • Venclexta

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
28 days
Treatment related toxicities
Time Frame: 60 days
Number of related adverse events
60 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Disease response
Time Frame: 42 days
Estimate of overall response rate (ORR) defined as (CR/CRi/CRp).
42 days
Cancer therapeutics-related cardiac dysfunction (CTRCD) in patients who have previously received anthracyclines
Time Frame: 60 days
Measured by echocardiogram (ECHO)
60 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Hospital Medical Center, Cincinnati

Investigators

  • Principal Investigator: John Perentesis, MD, Children's Hospital Medical Center, Cincinnati

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 27, 2018

Primary Completion (Estimated)

January 1, 2027

Study Completion (Estimated)

January 1, 2028

Study Registration Dates

First Submitted

January 9, 2019

First Submitted That Met QC Criteria

January 31, 2019

First Posted (Actual)

February 1, 2019

Study Record Updates

Last Update Posted (Actual)

February 18, 2026

Last Update Submitted That Met QC Criteria

February 16, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • V2-MA-1801

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Leukemia

Clinical Trials on Vyxeos

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Dominican Republic

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe