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

NCT ID: NCT03826992

Last Updated: 2025-05-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-12-27

Study Completion Date

2028-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Leukemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Group Type EXPERIMENTAL

Vyxeos

Intervention Type 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.

Venetoclax

Intervention Type 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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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.

Intervention Type 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

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

cytarabine and daunorubicin liposome, CPX-351 Venclexta

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are 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
Minimum Eligible Age

1 Year

Maximum Eligible Age

39 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

John Perentesis, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

United States

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Site Pulblic Contact

Role: CONTACT

513-636-2799

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Site Public Contact

Role: primary

513-636-2799

Related Links

Access external resources that provide additional context or updates about the study.

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

V2-MA-1801

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.