Actively Recruiting

Phase 1
Phase 2
Age: 18Years +
All Genders
NCT04128748

Liposomal Cytarabine and Daunorubicin (CPX-351) and Quizartinib for the Treatment of Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome

Led by M.D. Anderson Cancer Center · Updated on 2026-04-24

52

Participants Needed

1

Research Sites

396 weeks

Total Duration

On this page

Sponsors

M

M.D. Anderson Cancer Center

Lead Sponsor

N

National Cancer Institute (NCI)

Collaborating Sponsor

AI-Summary

What this Trial Is About

This phase I/II trial studies the side effects and best dose of CPX-351 in combination with quizartinib for the treatment of acute myeloid leukemia and high risk myelodysplastic syndrome. CPX-351, composed of chemotherapy drugs daunorubicin and cytarabine, works in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Quizartinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. The goal of this study is to learn if the combination of CPX-351 and quizartinib can help to control acute myeloid leukemia and myelodysplastic syndrome.

CONDITIONS

Official Title

Liposomal Cytarabine and Daunorubicin (CPX-351) and Quizartinib for the Treatment of Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of either acute myeloid leukemia (AML) with \u2265 20% blasts excluding acute promyelocytic leukemia or high-risk myelodysplastic syndrome (MDS) with >10% bone marrow blasts
  • Patients aged 60 years or older for frontline treatment cohort
  • Patients aged 18 years or older for relapsed or refractory cohort
  • Frontline patients must be chemotherapy-naive except limited use of hydroxyurea or 1-2 doses of ara-C for transient control
  • Relapsed or refractory patients may have received at least one prior therapy and up to four salvage regimens
  • At least 2 weeks since prior cytotoxic treatment or 5 half-lives of prior therapy before starting study treatment
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
  • Kidney function with creatinine < 1.8 mg/dl
  • Liver function with total bilirubin < 1.8 mg/dL unless due to hemolysis or congenital disorder
  • Transaminases less than 2.5 times the upper limit of normal
  • Normal potassium, magnesium, and calcium levels after correction
  • Ability to take oral medications
  • Ability to understand and sign informed consent
  • Baseline heart function with left ventricular ejection fraction (LVEF) ≥ 50%
  • Women of childbearing potential must have negative pregnancy test within 7 days
  • Men must agree to use condoms if partner is of childbearing potential
  • Women of childbearing potential must use effective contraception during and for 30 days after treatment
  • Patients may participate in supportive care trials but not other investigational cancer treatments
Not Eligible

You will not qualify if you...

  • Known allergy or hypersensitivity to quizartinib, mannitol, CPX-351, or their components
  • Electrolyte abnormalities: potassium < 3.5 mEq/L despite supplementation or > 5.5 mEq/L; magnesium or calcium outside normal limits despite management
  • Significant gastrointestinal conditions affecting quizartinib absorption
  • Severe or uncontrolled medical conditions including uncontrolled diabetes, cardiovascular disease, renal disease, or active infection
  • Active antineoplastic or radiation therapy for concurrent cancer (except maintenance, hormonal, or steroid therapy for well-controlled malignancy)
  • Known HIV infection
  • Positive hepatitis B or C infection unless viral load undetectable within 3 months
  • Consumption of grapefruit or related products within 3 days prior to treatment
  • Major surgery within 14 days before starting treatment
  • Cardiac issues: QTc interval > 450 msec, congenital long QT syndrome, history of serious arrhythmias, heart block (unless pacemaker present), slow heart rate < 50/min, specific bundle branch blocks
  • Recent myocardial infarction or unstable angina within 6 months
  • Congestive heart failure New York Heart Association class III or IV
  • Atrial fibrillation within 2 weeks before treatment
  • Use of strong CYP3A4 inducers
  • Requirement for drugs that prolong QT interval unless essential and approved by investigator
  • Family history of congenital long QT syndrome

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

M D Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

M

Musa Yilmaz

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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