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Acute Myeloid LeukemiaPhase 1/2NCT03793478

Safety and Efficacy of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML), a Cancer of the Blood

A Phase 1/2 study testing Quizartinib, Fludarabine and Cytarabine in people with Acute Myeloid Leukemia, run by Daiichi Sankyo at 27 sites in 10 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

Quizartinib is an experimental drug. It is not approved for regular use. It can only be used in medical research. Children or young adults with a certain kind of blood cancer (FLT3-ITD AML) might be able to join this study if it has come back after remission or is not responding to treatment. Read more about this study

Who is it for?
Ages 1 month to 21 years
Conditions the study is for
Acute Myeloid Leukemia
What is being tested?
Quizartinib, Fludarabine and Cytarabine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
65 participants
Where
27 sitesin 10 countries
Study dates
15 Aug 2018to 1 May 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 1 month to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Participants must meet all of the following criteria to be eligible for enrollment into the study:
  • Has diagnosis of AML according to the World Health Organization (WHO) 2008 classification with ≥5% blasts in bone marrow, with or without extramedullary disease
  • In first relapse or refractory to first-line high-dose chemotherapy with no more than 1 attempt (1 to 2 cycles of induction chemotherapy) at remission induction - prior HSCT is permitted
  • Has presence of the FLT3-ITD activating mutation in bone marrow or peripheral blood as defined in the protocol
  • Is between 1 month and 21 years of age at the time the Informed Consent/Assent form is signed
  • Has protocol-defined adequate performance status score
Show all 12Show fewer
  • Has fully recovered from the acute clinically significant toxicity effects of all prior chemotherapy, immunotherapy, or radiotherapy, per protocol guidelines
  • Has protocol-defined adequate renal, hepatic and cardiac functions
  • If of reproductive potential, is permanently sterile or agrees to use highly effective birth control upon enrollment, during the period of therapy, and for 6 months following the last dose of quizartinib, etoposide, fludarabine, methotrexate, or cytarabine, whichever is later
  • If female of child-bearing potential, tests negative for pregnancy and agrees not to breast feed
  • Male participants must be surgically sterile or willing to use highly effective birth control during the treatment period, and for 6 months following the last dose of quizartinib, etoposide, fludarabine, methotrexate, or cytarabine, whichever is later.
  • Participant/legal representative is capable of understanding the investigational nature of the study, potential risks, and benefits, and the patient (and/or legal representative) signs a written assent/informed consent

Who cannot join

  • Participants who meet any of the following criteria will be disqualified from entering the study:
  • Has been diagnosed with isolated central nervous system relapse, acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia, French-American-British classification M3 or WHO classification of APL with translocation, or with myeloid proliferations related to Down syndrome
  • Has uncontrolled or pre-defined significant cardiovascular disease as detailed in the protocol
  • Has systemic fungal, bacterial, viral or other infection that is exhibiting ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics or other treatment. The patient must be off vasopressors and have negative blood cultures for at least 48 hours prior to the start of systematic protocol therapy.
  • Has known active clinically relevant liver disease (e.g., active hepatitis B or active hepatitis C)
  • Has known history of human immunodeficiency virus (HIV)
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  • Has history of hypersensitivity to any of the study medications or their excipients
  • Is receiving or is anticipated to receive concomitant chemotherapy, radiation, or immunotherapy other than as specified in the protocol
  • Has any significant concurrent disease, illness, psychiatric disorder or social issue that would compromise subject safety or compliance, interfere with consent/assent, study participation, follow up, or interpretation of study results
  • Is currently participating in another investigative interventional procedure (observational or long-term interventional follow-up is allowed)
  • Is otherwise considered inappropriate for the study by the Investigator

Trial locations and contacts

27 sites in 10 countries.

27 sites in 10 countries

United States11 sites

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • The University of Texas Southwestern Medical Center Children's Health

    Dallas, Texas, 75390

  • Loma Linda University Cancer Center

    Loma Linda, California, 92354

  • University of Minnesota/Masonic Cancer Center

    Minneapolis, Minnesota, 55455

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224

  • University of California, San Francisco

    San Francisco, California, 94158

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

  • A.I. duPont Hospital for Children

    Wilmington, Delaware, 19803

Other trials in United States

France3 sites

  • Centre Léon Bérard

    Lyon, 69008

  • Hôpital Armand-Trousseau

    Paris, 75012

  • Hôpital des Enfants

    Toulouse, 31300

Other trials in France

Italy3 sites

  • Fondazione IRCCS San Gerardo dei Tintori

    Monza, 20900

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Rome, 00165

  • Ospedale Infantile Regina Margherita

    Torino, 10126

Other trials in Italy

Canada2 sites

  • The Hospital for Sick Children

    Toronto, Ontario, M5G1X8

  • British Columbia Children's Hospital

    Vancouver, V6H 3V4

Other trials in Canada

Israel2 sites

  • Rambam Medical Center

    Haifa, 31096

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 64239

Other trials in Israel

Spain2 sites

  • Hospital Infantil Universitario Nino Jesus

    Madrid, 28009

  • Hospital Universitario La Paz

    Madrid, 28046

Other trials in Spain

Belgium1 site

  • Universitair Ziekenhuis Gent

    Ghent

Other trials in Belgium

Denmark1 site

  • Rigshospitalet

    Copenhagen, 2100

Other trials in Denmark

Netherlands1 site

  • Prinses Maxima Centrum voor Kinderoncologie

    Utrecht, 3584 EA

Other trials in Netherlands

Sweden1 site

  • Sahlgrenska Universitetssjukhuset - Drottning Silvias Barn- och Ungdomssjukhus

    Gothenburg, 41685

Other trials in Sweden

About this study

Official title: A Phase 1/2, Multicenter, Dose-Escalating Study To Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy Of Quizartinib Administered in Combination With Re-Induction Chemotherapy, and as a Single-Agent Continuation Therapy, in Pediatric Relapsed/Refractory AML Subjects Aged 1 Month to <18 Years (and Young Adults Aged up to 21 Years) With FLT3-ITD Mutations

Quizartinib is an experimental drug. It is not approved for regular use. It can only be used in medical research.

Children or young adults with a certain kind of blood cancer (FLT3-ITD AML) might be able to join this study if it has come back after remission or is not responding to treatment.

Read the detailed description

The medical condition being investigated is relapsed or refractory AML in participants aged ≥1 month to ≤21 years with Feline McDonough Sarcoma (FMS)-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) mutations (FLT3-ITD AML), following failure of front-line intensive chemotherapy.

The trial will be conducted in multiple phases. An independent data monitoring committee (DMC) will protect the rights, safety, and well-being of participants by monitoring the progress and results. The DMC will comprise qualified physicians and scientists who are not Investigators in the study and not otherwise directly associated with the Sponsor and will be convened at the end of Phase 1.

  1. Dose Escalation/De-escalation Phase:

Number of participants is determined by age group. Participants will be enrolled by dose-level to determine the recommended Phase 2 dose (RP2D) of quizartinib for pediatric participants that provides similar exposure to adult patients treated at the target adult dose of 60 mg orally once daily.

  1. Dose-Expansion Phase:

Participants will receive the RP2D of quizartinib for their respective age group.

During both dose escalation and dose expansion phases, participants will receive:

Re-Induction Therapy

  • Intrathecal (IT) triple chemotherapy prophylaxis prior to and between cycles
  • In re-induction Cycles 1 and 2, fludarabine/cytarabine (FLA) followed by quizartinib as a single agent

Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) Period:

After re-induction therapy, participants will be evaluated for eligibility to undergo allogeneic hematopoietic stem cell transplant (HSCT). Eligible participants may receive a single 28-day cycle of consolidation therapy (standard of care chemotherapy with or without quizartinib) if an allogeneic HSCT is not available immediately. The options for consolidation therapy are as follows:

  • High intensity chemotherapy with quizartinib, or
  • Low intensity chemotherapy alone, or
  • Low intensity therapy with quizartinib as a single agent

Continuation Therapy:

Participants in remission after HSCT, or who are not eligible for HSCT but achieve at least a partial remission (PR) after re-induction, will receive up to 12 continuous 28-day cycles of quizartinib continuation therapy at the same dose received during re-induction in the dose expansion phase.

Long-term Follow-up:

The long-term follow-up phase begins upon completion of 12 cycles of quizartinib Continuation Therapy or permanent discontinuation of quizartinib at any time. After completion of the 30-day safety follow-up visit, subsequent visits will occur at the following frequencies to assess survival and anti-leukemic treatments:

  • every 3 months for the first 2 years, and then
  • once a year thereafter until the last participant enrolled has been followed for three years from the date of enrollment
Being tested
Quizartinib, Fludarabine, Cytarabine, Intrathecal (IT) triple chemotherapy prophylaxis, Etoposide
Keywords
Acute myeloid leukemia recurrent, Relapsed or refractory, FMS-like tyrosine kinase 3 positive, Cancer of the blood, AML, FLT3-ITD mutation
Medical subject headings
Leukemia, Myeloid, Acute, Recurrence

What participants receive

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

  • QuizartinibDrug

    Also called Quizartinib dihydrochloride, Vanflyta

    Administered orally once daily starting on Day 6 and continuing through Day 28;

    Optional low intensity consolidation with chemotherapy:

    Administered orally once daily starting on Day 1 and continuing through Day 28

  • FludarabineDrug

    30 mg/m^2/day IV infusion given over 30 minutes on Days 1 through 5 (administered based on body weight)

  • CytarabineDrug

    2000 mg/m^2/day IV infusion given over 3 hours on Days 1 through 5 (begin 4 hours after the start of fludarabine) (administered in accordance with standard of care);

    Optional high intensity consolidation with chemotherapy and quizartinib:

    500 mg/m^2/day as a continuous 96-hour IV infusion on Days 1 through 4;

    Optional low intensity consolidation with chemotherapy:

    75 mg/m^2/day as once daily subcutaneous or IV on Days 1 through 4 and Days 15 through 18

  • Intrathecal (IT) triple chemotherapy prophylaxisDrug

    IT cytarabine, methotrexate, and either prednisolone or hydrocortisone; doses are based on the participant's age and standard practice at each site

  • EtoposideDrug

    Optional high intensity consolidation with chemotherapy and quizartinib:

    100 mg/m^2/dose once daily as an IV infusion over 3 hours on Days 1 through 5

Study groups in NCT03793478
Study groupRoleWhat they receive
All ParticipantsExperimentalAll participants will receive re-induction therapy that includes fludarabine and cytarabine in combination with experimental drug quizartinib. For prophylaxis, IT chemotherapy with cytarabine, methotrexate and prednisolone/hydrocortisone will be given prior to or between re-induction cycles. After completing re-induction therapy, eligible participants may also receive optional consolidation chemotherapy which includes cytarabine, etoposide and quizartinib, if HSCT is not available immediately. After completing re-induction or HSCT successfully, eligible participants can go on to receive continuation therapy with quizartinib.
Receives: Drug: Quizartinib; Drug: Fludarabine; Drug: Cytarabine; Drug: Intrathecal (IT) triple chemotherapy prophylaxis; Drug: Etoposide

What is being measured

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

  1. Number of dose-limiting toxicities (Phase 1)

    Measured: Re-induction Cycle 1 Day 1 up to Day 28 (each cycle is 28 days)

  2. Composite complete remission (CRc) rate among participants with acute myeloid leukemia (AML) (Phase 1 and 2)

    CRc rate is defined as percentage of participants with best response of complete remission (CR) or CR with incomplete hematological recovery (CRi) after completion of up to 2 re-induction cycles

    Measured: within 8 years, 8 months

  3. Pharmacokinetic parameter area under the concentration curve for quizartinib and AC886 (Phase 1 and 2)

    Measured: Re-induction Phase 1 and 2,Cycle 1: Days 6, 20:Predose, 1 hour (Phase 1 only), 2, 4, and 6 hours; Days 7 and 21:24 hours; Phase 1 and 2 Cycle 2: Days 6 and 20:predose, 3 hours; Continuation Cycle 1 Days 1 and 15:predose, 2-4 hours (each cycle, 28 days)

  4. Pharmacokinetic parameter apparent clearance (CL/F) for quizartinib and AC886 (Phase 1 and 2)

    Measured: Re-induction Phase 1 and 2,Cycle 1: Days 6, 20:Predose, 1 hour (Phase 1 only), 2, 4, and 6 hours; Days 7 and 21:24 hours; Phase 1 and 2 Cycle 2: Days 6 and 20:predose, 3 hours; Continuation Cycle 1 Days 1 and 15:predose, 2-4 hours (each cycle, 28 days)

  5. Pharmacokinetic parameter apparent volume of distribution (Vz/F) for quizartinib and AC886 (Phase 1 and 2)

    Measured: Re-induction Phase 1 and 2,Cycle 1: Days 6, 20:Predose, 1 hour (Phase 1 only), 2, 4, and 6 hours; Days 7 and 21:24 hours; Phase 1 and 2 Cycle 2: Days 6 and 20:predose, 3 hours; Continuation Cycle 1 Days 1 and 15:predose, 2-4 hours (each cycle, 28 days)

Secondary outcomes (16)

  1. Complete remission (CR) rate among participants with AML (Phase 1 and 2)

    CR rate is defined as the percentage of participants achieving CR after completion of up to 2 re-induction cycles

    Measured: on Day 56 (± 3 Days) for the last subject, within 4 years

  2. Complete remission with incomplete recovery (CRi) rate among participants with AML (Phase 1 and 2)

    CRi rate is defined as the percentage of participants achieving CRi after completion of up to 2 re-induction cycles

    Measured: on Day 56 (± 3 Days) for the last subject, within 4 years

  3. Duration of CR among participants with AML (Phase 1 and 2)

    Duration of CR is defined as the time from the first documented CR until documented relapse

    Measured: within 8 years, 8 months

  4. Duration of CRi among participants with AML (Phase 1 and 2)

    Duration of CRi is defined as the time from the first documented CRi until documented relapse

    Measured: within 8 years, 8 months

  5. Duration of composite complete remission (CRc) among participants with AML (Phase 1 and 2)

    Duration of CRc is defined as the time from the first documented CR or CRi until documented relapse

    Measured: within 8 years, 8 months

  6. Complete remission (CR) rate after completion of re-induction Cycle 1 among participants with AML (Phase 1 and 2)

    CR rate after completion of re-induction Cycle 1 is defined as the percentage of participants achieving a CR after completion of re-induction Cycle 1

    Measured: on Day 56 (± 3 Days) for the last subject, within 4 years

  7. Complete remission with incomplete recovery (CRi) rate after completion of re-induction Cycle 1 among participants with AML (Phase 1 and 2)

    CRi rate after completion of re-induction Cycle 1 is defined as the percentage of participants achieving a CRi after completion of re-induction Cycle 1

    Measured: on Day 56 (± 3 Days) for the last subject, within 4 years

  8. Composite complete remission (CRc) rate after completion of re-induction Cycle 1 among participants with AML (Phase 1 and 2)

    CRc rate after completion of re-induction Cycle 1 is defined as the percentage of participants achieving CRc after completion of re-induction Cycle 1

    Measured: on Day 56 (± 3 Days) for the last subject, within 4 years

  9. Time to relapse among participants with AML (Phase 1 and 2)

    Time to relapse is defined as the time from the first documented response (CR, CRi) until documented relapse

    Measured: within 8 years, 8 months

  10. Rate of relapse among participants with AML after 1, 2 and 3 years (Phase 1 and 2)

    Rate of relapse is defined as the percentage of participants who achieved CRc at the end of re-induction and had relapsed at 3 categorical time points Categories: 1 year, 2 years, 3 years

    Measured: within 8 years, 8 months

  11. Cumulative incidence of relapse among participants with AML at the end of study (Phase 1 and 2)

    Cumulative incidence of relapse at the end of the study is defined as the percentage of participants who achieved CRc at the end of re-induction and relapsed by the end of the study

    Measured: within 8 years, 8 months

  12. Overall survival among participants with AML at 1, 2, and 3 years (Phase 1 and 2)

    Overall survival is defined as the time from the start of re-induction therapy until death from any cause

    Measured: within 8 years, 8 months

  13. Event-free survival among participants with AML at 1, 2, and 3 years (Phase 1 and 2)

    Event-free survival is defined as the time from the start of re-induction therapy until the earliest date of the following:

    • Refractory disease at the end of re-induction
    • Relapse after CR or CRi
    • Death from any cause at any time during the study

    Measured: within 8 years, 8 months

  14. Number of participants proceeding to high-dose conditioning therapy/allogeneic hematopoietic stem cell transplantation (HSCT) (including transplant-related mortality) among participants with AML (Phase 1 and 2)

    Measured: within 8 years, 8 months

  15. Rate of CRc (CR or CRi) without minimal residual disease (MRD) using next generation sequencing among participants with AML (Phase 1 and 2)

    MRD is defined as the presence of leukemic cells in the bone marrow detected above a predefined cut-off level by a validated assay in participants who achieved a CR or CRi

    Categories: at screening, at re-induction cycle 1, at re-induction cycle 2, at time of relapse

    Measured: within 8 years, 8 months

  16. Acceptability of including the palatability of quizartinib formulations among participants with AML (Phase 1 and 2)

    Acceptability is assessed by palatability using a 5-point hedonic scale (from "Super bad" to "Super good") for participants who are developmentally able to answer. If unable, the caregiver provides information in a free text field stating whether the participant spit it out, may not have liked the flavor, etc.

    Measured: within 8 years, 8 months

Timeline

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

  1. First posted4 Jan 2019
  2. Study start15 Aug 2018
  3. Primary completion1 May 2027 (estimated)
  4. Study completion1 May 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
Sequential
Model description
A single group will progress through a multiple phase study design as described in the detailed description.
Masking
None (open label)
Enrolment
65 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03793478
Sponsor's protocol ID
AC220-A-U202
Other IDs
2016-002919-18, 2016
Status last verified
Mar 2026
First submitted
2 Jan 2019
Last updated
1 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Quizartinib, Fludarabine and Cytarabine in people with Acute Myeloid Leukemia, run by Daiichi Sankyo at 27 sites in 10 countries.

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 12 inclusion and 11 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: under way, not looking for participants, as of the sponsor's last update on 1 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 27 sites in United States, France, Italy, Canada, Israel and 5 more countries. 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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of dose-limiting toxicities (Phase 1) (time frame: Re-induction Cycle 1 Day 1 up to Day 28 (each cycle is 28 days)). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03793478.

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 1 Apr 2026; AzTrials last refreshed its copy on 8 Nov 2026.

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