LeukemiaPhase 1NCT03826992
Venetoclax Combined With Vyxeos (CPX-351) for Participants With Relapsed or Refractory Acute Leukemia
A Phase 1 study testing Vyxeos and Venetoclax in people with Leukemia, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 39 years
- Conditions the study is for
- Leukemia
- What is being tested?
- Vyxeos and Venetoclax
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Dec 2018to Jan 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 39 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Site Public Contact, 513-636-2799, cancer@cchmc.org
John Perentesis, MD
About this study
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
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.
Read the 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.
- Conditions
- Leukemia
- Being tested
- Vyxeos, Venetoclax
- 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)
- Medical subject headings
- Leukemia, Recurrence, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VyxeosDrug
Also called cytarabine and daunorubicin liposome, CPX-351
Vyxeos Dose: daunorubicin 44 mg/m2 and cytarabine 100 mg/m2 administered via intravenous infusion over 90 minutes on Days 1, 3, and 5.
VenetoclaxDrug
Also called Venclexta
Venetoclax Dose:
- Dose Level 0 - weight based daily dosing for 21 days
- Dose Level -1 - weight based daily dosing for 14 days
- Dose Level -2- weight based daily dosing for 10 days
- Dose Level -3- weight based daily dosing for 7 days
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Vyxeos; Drug: Venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 28 days
Treatment related toxicities
Number of related adverse events
Measured: 60 days
Secondary outcomes (2)
Disease response
Estimate of overall response rate (ORR) defined as (CR/CRi/CRp).
Measured: 42 days
Cancer therapeutics-related cardiac dysfunction (CTRCD) in patients who have previously received anthracyclines
Measured by echocardiogram (ECHO)
Measured: 60 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2019
- Study start27 Dec 2018
- Primary completionJan 2027 (estimated)
- Study completionJan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- John Perentesis, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03826992
- Sponsor's protocol ID
- V2-MA-1801
- Status last verified
- Feb 2026
- First submitted
- 9 Jan 2019
- Last updated
- 18 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03826992
- Acronym / sponsor ID
- V2-MA-1801
- Conditions
- Leukemia
- Keywords
- relapsed, refractory, Vyxeos, Venetoclax, acute myeloid leukemia, acute myeloid leukemia, childhood, mixed-lineage leukemia (MLL), AML
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- 27 Dec 2018 (actual)
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Jan 2028 (estimated)
- First posted
- 1 Feb 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Venetoclax and Vyxeos combination | Experimental | Drug: Vyxeos, Drug: VenetoclaxVenetoclax 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. |
Who may qualify
- Ages
- 1 year to 39 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (24 listed)
- Ages 1 Year to 39 Years
- Diagnosis of one of the following:
- Disease Status
- Karnofsky/Lanksy performance level score of greater than or equal to 50 percent.
- Prior therapy requirements
Key exclusion criteria (14 listed)
- Diagnosis of one of the following:
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VyxeosAlso: cytarabine and daunorubicin liposome, CPX-351Vyxeos Dose: daunorubicin 44 mg/m2 and cytarabine 100 mg/m2 administered via intravenous infusion over 90 minutes on Days 1, 3, and 5. | Drug | Venetoclax and Vyxeos combination |
| VenetoclaxAlso: VenclextaVenetoclax 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 | Drug | Venetoclax and Vyxeos combination |
Primary outcome
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
2 secondary outcomes
Disease response
Time frame: 42 days
Cancer therapeutics-related cardiac dysfunction (CTRCD) in patients who have previously received anthracyclines
Time frame: 60 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Feb 2026.
- Cincinnati Children's Cancer and Blood Diseases InstituteLink provided by the sponsor.
- Cincinnati Children's Hospital Oncology DivisionLink provided by the sponsor.
- Leukemia and Lymphoma ProgramLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
John Perentesis, MD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Vyxeos and Venetoclax in people with Leukemia, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 24 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Site Pulblic Contact (513-636-2799, cancer@cchmc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Feasibility of combining venetoclax and Vyxeos (dose limiting toxicities) (time frame: 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Feb 2026.