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

NCT03826992 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: V2-MA-1801
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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

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)

Outcome Measures

Primary Outcomes (2)

• 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.

  28 days

• Treatment related toxicities

  Number of related adverse events

  60 days

Secondary Outcomes (2)

• Disease response

  42 days

• Cancer therapeutics-related cardiac dysfunction (CTRCD) in patients who have previously received anthracyclines

  60 days

Study Arms (1)

Venetoclax and Vyxeos combination

EXPERIMENTAL

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

Interventions

Vyxeos DRUG

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 and Vyxeos combination

Venetoclax DRUG

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

Venetoclax and Vyxeos combination

Eligibility Criteria

• Age: 1 Year - 39 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Related Links

• Cincinnati Children's Cancer and Blood Diseases Institute
• Cincinnati Children's Hospital Oncology Division
• Leukemia and Lymphoma Program

MeSH Terms

Conditions

Leukemia; Recurrence; Leukemia, Myeloid, Acute

Interventions

Cytarabine; Daunorubicin; CPX-351; venetoclax

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Leukemia, Myeloid

Intervention Hierarchy (Ancestors)

Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Anthracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds; Aminoglycosides; Glycosides; Carbohydrates

Study Officials

• John Perentesis, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Site Pulblic Contact

CONTACT

513-636-2799; cancer@cchmc.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 9, 2019
• First Posted: February 1, 2019
• Study Start: December 27, 2018
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2028
• Last Updated: February 18, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)