Fms-like tyrosine kinase 3 positive Trials in Disease

Study on the Safety and Effectiveness of Quizartinib for Children and Young Adults with Acute Myeloid Leukemia (AML)

This clinical trial is investigating Quizartinib, a targeted medication designed to treat acute myeloid leukemia in children and young adults who have a specific genetic mutation called FLT3-ITD. This mutation is found in some cases of this type of blood cancer, and Quizartinib is designed to specifically target and block the abnormal signals that help cancer cells grow and survive.

Who can participate:

• Children and young adults between 1 month and 21 years old
• Patients must have a confirmed diagnosis of acute myeloid leukemia with at least 5% abnormal cells in the bone marrow
• The cancer must have the FLT3-ITD genetic mutation present in bone marrow or blood
• Patients must be experiencing their first relapse (return of cancer) or have not responded to the first round of high-dose chemotherapy
• Adequate kidney, liver, and heart function is required
• Patients must have recovered from significant side effects of previous cancer treatments
• Those who can have children must agree to use highly effective birth control during the study and for 6 months after treatment ends
• Female patients must have a negative pregnancy test and agree not to breastfeed during the study

Who cannot participate:

• Patients with a different type of cancer other than acute myeloid leukemia
• Patients outside the specified age range
• Those who cannot safely receive the study medication or chemotherapy due to other health conditions
• Pregnant or breastfeeding patients
• Patients with certain infections or serious health issues that could interfere with the study
• Those unable to follow study procedures or attend required visits

What the trial involves:

The trial has several treatment phases. First, patients receive up to two cycles of re-induction treatment combining Quizartinib with chemotherapy. The goal during this phase is to achieve complete remission, meaning the cancer is no longer detectable. Quizartinib is given as an oral solution taken by mouth.

After the re-induction cycles, patients may receive optional consolidation therapy, which is additional chemotherapy to help maintain remission. Following this, patients transition to a continuation phase where they receive only Quizartinib without chemotherapy for up to 12 cycles. This aims to keep the cancer from returning.

Throughout the trial, doctors will closely monitor how well the treatment works in reducing cancer cells, how long remission lasts, and any side effects that occur. The study will also evaluate the best dose of Quizartinib for different age groups and how the body processes the medication.

Investigational drug:

Quizartinib is a type of medication known as a tyrosine kinase inhibitor. It works by blocking the FLT3 receptor, a protein that is involved in the growth and survival of cancer cells. By targeting this specific protein, Quizartinib aims to stop cancer cells from multiplying while causing less harm to normal cells. The medication is currently being studied in clinical trials and is not yet approved for widespread use outside of research settings.