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Acute Myeloid LeukemiaPhase 1/2NCT04047641

Cladribine, Idarubicin, Cytarabine, and Quizartinib in Treating Patients With Newly Diagnosed, Relapsed, or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

A Phase 1/2 study testing Cladribine, Cytarabine and Idarubicin in people with Acute Myeloid Leukemia and Blasts 20 Percent or More of Bone Marrow Nucleated Cells, run by M.D. Anderson Cancer Center at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

This phase I/II trial studies the side effects and how well cladribine, idarubicin, cytarabine, and quizartinib work in treating patients with acute myeloid leukemia or high-risk myelodysplastic syndrome that is newly diagnosed, has come back (relapsed), or does not respond to treatment (refractory). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Cladribine, Cytarabine and Idarubicin
Could I receive a placebo?
No, everyone receives an active treatment
How many people
80 participants
Where
1 siteUnited States
Study dates
22 Oct 2019to 31 Dec 2027
Phase
Phase 1/2
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Diagnosis of
  • AML (World Health Organization [WHO] classification definition of >= 20% blasts, excluding Acute promyelocytic leukemia),
  • Acute biphenotypic leukemia or
  • High-risk MDS (> 10% bone marrow blasts)
  • Frontline cohort: Patients aged 18 to 65 years
  • Relapse cohort: Patients aged >=18 years old
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  • Patients may be newly diagnosed (Frontline cohort) or with prior therapy (Relapsed cohort) as follows:
  • For frontline cohort: Patients must be chemonaive, i.e., not have received any chemotherapy (except hydroxyurea [Hydrea] [no dose limit], tretinoin [atra] [no dose limit] or ara-C [one or two doses (max 2 gr/m^2 per dose)] for transient control of hyperleukocytosis) for AML or MDS. They may have received hypomethylating agents for prior MDS and transfusions, hematopoietic growth factors or vitamins. Temporary prior measures such as apheresis or Hydrea are allowed
  • For relapsed cohort: Patients with previously treated, relapsed or refractory AML, acute biphenotypic leukemia or high-risk MDS (> 10% bone marrow blasts)
  • Eastern Cooperative Oncology Group (ECOG) performance status =< 2
  • Creatinine < 1.5 mg/dl
  • Total bilirubin < 1.5 mg/dL, unless increase is due to hemolysis or congenital disorder
  • Transaminases (serum glutamate pyruvate transaminase [SGPT]) < 2.5 x upper limit of normal (ULN)
  • Potassium, magnesium, and calcium (normalized for albumin) levels should be at least within institutional normal limits
  • Ability to take oral medication
  • Ability to understand and provide signed informed consent
  • Baseline test of left ventricular ejection fraction >= 50%
  • Women of childbearing potential (WOCBP) must have a negative serum or urine pregnancy test within 7 days
  • WOCBP must use appropriate method(s) of contraception such as oral contraceptive pills (OCP), birth control shots, intrauterine device (IUD) etc. WOCBP should use an adequate method to avoid pregnancy until 30 days after the last dose of investigational drug. Men must agree not to father a child and agree to use a condom if his partner is of child bearing potential. Women who are not of childbearing potential (ie, who are postmenopausal or surgically sterile) as well as men with known azoospermia do not require contraception
  • Patients with isolated extramedullary myeloid neoplasm will be eligible

Who cannot join

  • Any coexisting medical condition that in the judgment of the treating physician is likely to interfere with study procedures or results
  • Breastfeeding women
  • Patients with current active malignancies or any remission for < 6 months, except patients with carcinoma in situ or with non-melanoma skin cancer who may be in remission for less than 6 months or have active disease
  • Active clinically serious and uncontrolled infection. Patients with recent infections must have no temperature of >= 101 degrees Fahrenheit (F) for at least 48 hours (hrs) (before first dose, day 1)
  • Patients with known significant impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of quizartinib
  • Documented active central nervous system leukemia (patients with history of central nervous system [CNS] leukemia without active disease are allowed)
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  • Patients with a known confirmed diagnosis of human immunodeficiency virus (HIV) infection or active viral hepatitis
  • Patients who have had any major surgical procedure within 14 days of day 1
  • Impaired cardiac function including any of the following:
  • Screening electrocardiography (ECG) with a corrected QT (QTc) > 450 msec. The QTc interval will be calculated by Fridericia's correction factor (QTcF). The QTcF will be derived from the average QTcF in triplicate. Patients are excluded if they have QTcF >= 450. Subjects with prolonged QTcF interval in the setting of RBBB (right bundle branch block) may participate upon review and approval by the medical monitor. RBBB for patients' triplicate electrocardiograms (EKGs) can show false QTc prolongation; therefore, the cardiology collaborator for this study will manually review to provide an accurate reading of the QTc
  • Patients with congenital long QT syndrome
  • Sustained ventricular tachycardia requiring medical intervention
  • Any history of clinically significant ventricular fibrillation or torsades de pointes
  • Known history of second or third degree heart block (may be eligible if the patient currently has a pacemaker)
  • Heart rate of < 50/minute on pre-entry ECG
  • Left bundle branch block
  • Right bundle branch block + left anterior hemiblock (bifascicular block)
  • Patients with myocardial infarction or unstable angina within 6 months prior to starting study drug
  • Congestive heart failure (CHF) New York (NY) Heart Association class III or IV
  • Atrial fibrillation documented within 2 weeks prior to first dose of study drug
  • Known family history of congenital long QT syndrome
  • Patients who are actively taking a strong CYP3A4 inducing medication

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
  • M D Anderson Cancer Center

    Houston, Texas, 77030

    Musa Yilmaz, 713-794-5783

    Musa Yilmaz

    Recruiting

Other trials in United States

About this study

Official title: A Combination of Cladribine, Idarubicin, Cytarabine (CLIA) and Quizartinib for the Treatment of Patients With Newly Diagnosed or Relapsed/Refractory Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndrome (MDS))

This phase I/II trial studies the side effects and how well cladribine, idarubicin, cytarabine, and quizartinib work in treating patients with acute myeloid leukemia or high-risk myelodysplastic syndrome that is newly diagnosed, has come back (relapsed), or does not respond to treatment (refractory). Drugs used in chemotherapy, such as cladribine, idarubicin, and cytarabine, work 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 cancer cells by blocking some of the enzymes needed for cell growth. Giving quizartinib with cladribine, idarubicin, and cytarabine may help to control acute myeloid leukemia or high-risk myelodysplastic syndrome.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To determine the efficacy of quizartinib (AC220) in combination with cladribine, idarubicin and cytarabine (ara-C) induction chemotherapy in newly diagnosed or relapsed/refractory patients with high-risk myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML).

II. To determine the safety of the combination.

SECONDARY OBJECTIVES:

  1. To determine the overall survival and disease-free survival of patients treated with this combination.

II. To investigate correlations of response to this combination with a 81-gene panel of gene mutations both in patients with and without FLT3 mutations.

III. To identify individual treatment-resistant cell populations and their signaling state that may relate to clinical outcomes using CyTOF (cytometry by time of flight) and single cell sequencing.

OUTLINE:

INDUCTION: Patients receive idarubicin intravenously (IV) over 1 hour on days 1-3, cladribine IV over 1-2 hours on days 1-5, cytarabine IV over 2 hours on days 1-5 (or days 1-3 for patients over age 60), and quizartinib orally (PO) once daily (QD) on days 6-19. Treatment repeats every 28 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity.

CONSOLIDATION: Patients who achieve complete response (CR) or CR with incomplete platelet recovery (CRp) after Induction receive idarubicin IV over 1 hour on days 1-2, cladribine IV over 1-2 hours on days 1-3, cytarabine IV over 2 hours on days 1-3, and quizartinib PO QD on days 4-28. Treatment repeats every 28 days for up to 5 cycles in the absence of disease progression or unacceptable toxicity.

MAINTENANCE: Patients who achieve CR or CR with incomplete bone marrow recovery (CRi)/CR with partial hematologic recovery (CRh) after Consolidation receive quizartinib PO QD on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 6-12 months.

Being tested
Cladribine, Cytarabine, Idarubicin, Quizartinib
Medical subject headings
Leukemia, Myeloid, Acute, Leukemia, Biphenotypic, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CladribineDrug

    Also called 2-CdA, 2CDA, CdA, Cladribina

    Given Intravenous

  • CytarabineDrug

    Also called .beta.-Cytosine arabinoside, 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-.beta.-D-Arabinofuranosylcytosine, 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone

    Given Intravenous

  • IdarubicinDrug

    Also called 4-Demethoxydaunomycin, 4-demethoxydaunorubicin, 4-DMDR

    Given Intravenous

  • QuizartinibDrug

    Also called AC-220, AC010220, AC220

    Given by mouth

Study groups in NCT04047641
Study groupRoleWhat they receive
Treatment (idarubicin, cladribine, cytarabine, quizartinib)ExperimentalINDUCTION: Patients receive idarubicin Intravenous over 1 hours on days 1-3, cladribine intravenous over 1-2 hours on days 1-5, cytarabine Intravenous over 3 hours on days 1-5 (or days 1-3 for patients over age 60), and quizartinib by mouth daily on days 6-19. Treatment repeats every 28 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity. CONSOLIDATION: Patients who achieve CR or CRp after Induction receive idarubicin Intravenous over 1 hours on days 1-2, cladribine Intravenous over 1-2 hours on days 1-3, cytarabine Intravenous over 3 hours on days 1-3, and quizartinib by mouth daily on days 4-28. Treatment repeats every 28 days for up to 5 cycles in the absence of disease progression or unacceptable toxicity. MAINTENANCE: Patients who achieve CR or CRi/CRh after Consolidation receive quizartinib by mouth daily on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.
Receives: Drug: Cladribine; Drug: Cytarabine; Drug: Idarubicin; Drug: Quizartinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Event free survival (EFS)

    Will be estimate using the Kaplan-Meier method for each patient cohort. In addition, efficacy EFS will be analyzed in the intent to treat (ITT) population per cohort.

    Measured: From the date of start of treatment until event (resistance or relapse) or death, whichever occurred first, assessed up to 12 months

  2. Incidence of adverse events

    Defined as any clinically significant treatment-related grade 3 or greater non-hematologic toxicity. Patient toxicity data will be summarized using frequency and percentages, by type, grade and relationship to the study drugs.

    Measured: Up to 12 months

Secondary outcomes (5)
  1. Overall survival (OS)

    Will be estimate using the Kaplan-Meier method for each patient cohort. In addition, efficacy OS will be analyzed in the ITT population per cohort.

    Measured: Up to 12 months

  2. Disease free survival (DFS)

    Will be estimate using the Kaplan-Meier method for each patient cohort. In addition, efficacy DFS will be analyzed in the ITT population per cohort.

    Measured: Up to 12 months

  3. Rate of response

    The rate of response will be estimated for each 28-gene panel of gene mutations for each patient cohort.

    Measured: Up to 12 months

  4. Change of FLT3 ligand level

    Will also explore the change of FLT3 ligand level before and after treatment using summary statistics, by patient cohort and by other subgroups (i.e. age).

    Measured: Baseline up to 12 months

  5. Change in the presence of other gene mutations

    Will also explore the change in the presence of other gene mutations before and after treatment using summary statistics, by patient cohort and by other subgroups (i.e. age).

    Measured: Baseline up to 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Aug 2019
  2. Study start22 Oct 2019
  3. Primary completion31 Dec 2027 (estimated)
  4. Study completion31 Dec 2027 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
80 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04047641
Sponsor's protocol ID
2017-0153
Other IDs
NCI-2019-04730, 2017-0153
Status last verified
Jun 2026
First submitted
5 Aug 2019
Last updated
12 Jun 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Cladribine, Cytarabine and Idarubicin in people with Acute Myeloid Leukemia and Blasts 20 Percent or More of Bone Marrow Nucleated Cells, run by M.D. Anderson Cancer Center 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 20 inclusion and 22 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 12 Jun 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 Musa Yilmaz (713-794-5783, myilmaz@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Event free survival (EFS) (time frame: From the date of start of treatment until event (resistance or relapse) or death, whichever occurred first, assessed up to 12 months). 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 12 Jun 2026.

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