Last updated on August 2019

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


Brief description of study

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.

Detailed Study Description

The medical condition being investigated is relapsed or refractory AML in patients 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, using an adaptive trial design. 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.

  1. Dose Escalation/De-escalation Phase:

Cohorts of up to 9 participants per dose-level will be enrolled 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 once daily.

B. Dose-Expansion Phase:

Participants will receive the RP2D of quizartinib for their respective age group, administered in combination with re-induction chemotherapy

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

  • Intrathecal (IT) triple chemotherapy prophylaxis (given three times)
  • In re-induction Cycle 1, fludarabine/cytarabine (FLA) plus daunorubicin (liposomal daunorubicin preferred), followed by quizartinib
  • In re-induction Cycle 2, FLA followed by quizartinib as a single agent C. 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 might 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, as follows:

  • High intensity chemotherapy with quizartinib, or
  • Low intensity chemotherapy alone, or
  • Low intensity therapy with quizartinib as a single agent D. Maintenance:

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 maintenance therapy at the same dose received during re-induction in the dose expansion phase.

E. Long-term Follow-up:

The long-term follow-up phase begins upon completion of the 12 cycles of quizartinib Maintenance 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 yearly thereafter until the last participant enrolled has been followed for three years from the date of enrollment

Clinical Study Identifier: NCT03793478

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