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

  • STATUS
    Recruiting
  • End date
    May 1, 2027
  • participants needed
    65
  • sponsor
    Daiichi Sankyo, Inc.
Updated on 11 November 2020
Investigator
Daiichi Sankyo Contact for Clinical Trial Information
Primary Contact
Children's National Medical Center (5.4 mi away) Contact
+30 other location
cancer
remission
fludarabine
hematologic malignancy
methotrexate
cytarabine
induction chemotherapy
consolidation therapy
flt3 internal tandem duplication
blast cells

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.

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.

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

Details
Treatment cytarabine, etoposide, Fludarabine, Daunorubicin, Quizartinib, Intrathecal (IT) triple chemotherapy prophylaxis
Clinical Study IdentifierNCT03793478
SponsorDaiichi Sankyo, Inc.
Last Modified on11 November 2020

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Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age between 1 yrs and 21 yrs?
Gender: Male or Female
Do you have any of these conditions: Acute myeloid leukemia or Acute Myelogenous Leukemia (AML)?
Has diagnosis of AML according to the World Health Organization (WHO) 2008 classification with >5% blasts in bone marrow, with or without extramedullary disease
Is 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 study drug or cytarabine, whichever is later
If female of child-bearing potential, tests negative for pregnancy and agrees not to breast feed
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
Meets protocol-specified guidelines before inclusion in the continuation therapy phase

Exclusion Criteria

Has been diagnosed with isolated central nervous system relapse, certain kinds of leukemia, 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
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