Safety and Efficacy of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML), a Cancer of the Blood
NCT ID: NCT03793478
Last Updated: 2025-08-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
65 participants
INTERVENTIONAL
2018-08-15
2027-05-01
Brief Summary
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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.
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Detailed Description
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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.
A. 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.
B. 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
Conditions
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Study Design
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NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
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All Participants
All 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.
Quizartinib
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
Fludarabine
30 mg/m\^2/day IV infusion given over 30 minutes on Days 1 through 5 (administered based on body weight)
Cytarabine
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 prophylaxis
IT cytarabine, methotrexate, and either prednisolone or hydrocortisone; doses are based on the participant's age and standard practice at each site
Etoposide
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
Interventions
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Quizartinib
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
Fludarabine
30 mg/m\^2/day IV infusion given over 30 minutes on Days 1 through 5 (administered based on body weight)
Cytarabine
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 prophylaxis
IT cytarabine, methotrexate, and either prednisolone or hydrocortisone; doses are based on the participant's age and standard practice at each site
Etoposide
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
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
Exclusion Criteria
* 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)
* 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
1 Month
21 Years
ALL
No
Sponsors
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Innovative Therapies For Children with Cancer Consortium
OTHER
Children's Oncology Group
NETWORK
Daiichi Sankyo
INDUSTRY
Responsible Party
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Principal Investigators
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Global Clinical Leader
Role: STUDY_DIRECTOR
Daiichi Sankyo
Locations
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Loma Linda University Cancer Center
Loma Linda, California, United States
University of California, San Francisco
San Francisco, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
A.I. duPont Hospital for Children
Wilmington, Delaware, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
The University of Texas Southwestern Medical Center Children's Health
Dallas, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Universitair Ziekenhuis Gent
Ghent, , Belgium
The Hospital for Sick Children
Toronto, Ontario, Canada
British Columbia Children's Hospital
Vancouver, , Canada
Rigshospitalet
Copenhagen, , Denmark
Centre Léon Bérard
Lyon, , France
Hôpital Armand-Trousseau
Paris, , France
Hôpital des Enfants
Toulouse, , France
Rambam Medical Center
Haifa, , Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, , Israel
Fondazione IRCCS San Gerardo dei Tintori
Monza, , Italy
IRCCS Ospedale Pediatrico Bambino Gesù
Rome, , Italy
Ospedale Infantile Regina Margherita
Torino, , Italy
Prinses Maxima Centrum voor Kinderoncologie
Utrecht, , Netherlands
Hospital Infantil Universitario Nino Jesus
Madrid, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Sahlgrenska Universitetssjukhuset - Drottning Silvias Barn- och Ungdomssjukhus
Gothenburg, , Sweden
Countries
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Other Identifiers
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2016-002919-18
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
AC220-A-U202
Identifier Type: -
Identifier Source: org_study_id
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