Acute Myeloid Leukemia Clinical Trial

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

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.

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Full 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.

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

View Eligibility Criteria

Eligibility Criteria

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
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

Study is for people with:

Acute Myeloid Leukemia

Phase:

Phase 1

Estimated Enrollment:

65

Study ID:

NCT03793478

Recruitment Status:

Recruiting

Sponsor:

Daiichi Sankyo, Inc.

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There are 36 Locations for this study

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Loma Linda University Cancer Center
Loma Linda California, 92354, United States More Info
Study Coordinator
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University of California, San Francisco
San Francisco California, 94158, United States More Info
Study Coordinator
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Children's Hospital Colorado
Aurora Colorado, 80045, United States More Info
Study Coordinator
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A.I. duPont Hospital for Children
Wilmington Delaware, 19803, United States More Info
Study Coordinator
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Children's National Medical Center
Washington District of Columbia, 20010, United States More Info
Study Coordinator
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Children's Healthcare of Atlanta
Atlanta Georgia, 30322, United States More Info
Study Coordinator
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Riley Hospital for Children - Indiana University
Indianapolis Indiana, 46202, United States More Info
Study Coordinator
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Johns Hopkins
Baltimore Maryland, 21287, United States More Info
Study Coordinator
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University of Minnesota/Masonic Cancer Center
Minneapolis Minnesota, 55455, United States More Info
Site Coordinator
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University of Mississippi Medical Center
Jackson Mississippi, 39216, United States More Info
Site Coordinator
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Washington University School of Medicine
Saint Louis Missouri, 63110, United States More Info
Study Coordinator
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Columbia University/Herbert Irving Cancer Center
New York New York, 10032, United States More Info
Study Coordinator
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New York Medical College
Valhalla New York, 10595, United States More Info
Site Coordinator
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Cincinnati Children's Hospital Medical Center
Cincinnati Ohio, 45229, United States More Info
Study Coordinator
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Children's Hospital of Philadelphia
Philadelphia Pennsylvania, 19104, United States More Info
Study Coordinator
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UPMC Children's Hospital of Pittsburgh
Pittsburgh Pennsylvania, 15224, United States More Info
Study Coordinator
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The University of Texas Southwestern Medical Center Children's Health
Dallas Texas, 75390, United States More Info
Study Coordinator
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Seattle Children's Hospital
Seattle Washington, 98105, United States More Info
Study Coordinator
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Universitair Ziekenhuis Gent
Gent , , Belgium More Info
Principal Investigator
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The Hospital for Sick Children
Toronto Ontario, M5G1X, Canada More Info
Site Coordinator
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Montreal Children's Hospital
Montréal Quebec, H4A3J, Canada More Info
Study Coordinator
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British Columbia Children's Hospital
Vancouver , V6H 3, Canada More Info
Site Coordinator
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Rigshospitalet
Copenhagen , 2100, Denmark More Info
Principal Investigator
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Centre Léon Bérard
Lyon , 69008, France More Info
Principal Investigator
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Hôpital Armand-Trousseau
Paris , 75012, France More Info
Principal Investigator
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Hôpital des Enfants
Toulouse , 31300, France More Info
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Rambam Medical Center
Haifa , 31096, Israel More Info
Principal Investigator
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Schneider Children's Medical Center of Israel
Petah Tikva , 49202, Israel
Tel Aviv Sourasky Medical Center
Tel Aviv-Yafo , 64239, Israel More Info
Principal Investigator
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Fondazione MBBM - Clinica Pediatrica
Monza , 20900, Italy More Info
Principal Investigator
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IRCCS Ospedale Pediatrico Bambino Gesù
Rome , 00165, Italy More Info
Principal Investigator
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Ospedale Infantile Regina Margherita
Torino , 10126, Italy More Info
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Prinses Maxima Centrum voor Kinderoncologie
Utrecht , 3584 , Netherlands More Info
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Hospital Infantil Universitario Nino Jesus
Madrid , 28009, Spain More Info
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Hospital Universitario La Paz
Madrid , 28046, Spain More Info
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Sahlgrenska Universitetssjukhuset - Drottning Silvias Barn- och Ungdomssjukhus
Göteborg , 41685, Sweden More Info
Principal Investigator
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NHS Greater Glasgow and Clyde - The Queen Elizabeth University Hospital
Glasgow , G51 4, United Kingdom More Info
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How clear is this clinincal trial information?

Study is for people with:

Acute Myeloid Leukemia

Phase:

Phase 1

Estimated Enrollment:

65

Study ID:

NCT03793478

Recruitment Status:

Recruiting

Sponsor:


Daiichi Sankyo, Inc.

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