Recruiting
Phase 1

Venetoclax & Vyxeos

Sponsor:

Children's Hospital Medical Center, Cincinnati

Code:

NCT03826992

Conditions

Leukemia

Eligibility Criteria

Sex: All

Age: 1 - 39

Healthy Volunteers: Not accepted

Interventions

Vyxeos

Venetoclax

Study Details

Brief summary:

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.

Conditions

Leukemia

Study ID

NCT03826992

Start date

Dec 27, 2018

Status verified date

Feb, 2026

Completion date

Jan, 2028

Anticipated

Primary completion date

Jan, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1 - 39

Healthy Volunteers: Not accepted

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 Design

Enrollment

21 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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.

Interventions

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.

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 measure

  • Feasibility of combining venetoclax and Vyxeos (dose limiting toxicities) [ Time Frame: 28 days ]
  • Treatment related toxicities [ Time Frame: 60 days ]

Central Contacts and Locations

Central contacts

Site Pulblic Contact

513-636-2799cancer@cchmc.org

Locations

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Site Public Contact

513-636-2799cancer@cchmc.org

Principal Investigator:

John Perentesis, MD

More Information

Sponsor

Children's Hospital Medical Center, Cincinnati

Last update posted

Feb 18, 2026

Last verified

Feb, 2026

Keywords

  • 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)

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Children's Hospital Medical Center, Cincinnati on 2026-02-18.