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

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What is this study about?

This clinical trial is focused on studying a type of blood cancer called Acute Myeloid Leukemia (AML), which affects children and young adults. The study is investigating a treatment using a medication called Quizartinib, also known by its code name AC220. Quizartinib is a type of drug known as a Class III receptor tyrosine kinase inhibitor, which is designed to target specific mutations in cancer cells. The trial aims to evaluate the safety and effectiveness of Quizartinib when used in combination with chemotherapy and as a standalone treatment.

The purpose of the study is to determine the best dose of Quizartinib for different age groups and to assess how well it works in achieving remission, which means the cancer is no longer detectable. The study will also look at the safety of the treatment and how the body processes the medication. Participants will receive Quizartinib along with chemotherapy for up to two cycles, with the option for additional treatment if needed. After this, Quizartinib may be given alone for up to twelve cycles to see if it helps keep the cancer from returning.

This trial is specifically for patients with a mutation called FLT3-ITD, which is found in some cases of AML. The study will monitor how well the treatment works in reducing cancer cells and how long the remission lasts. It will also track any side effects experienced by participants. The goal is to find a safe and effective treatment option for young patients with this type of leukemia.

1 joining the study

Upon joining the study, the patient is confirmed to have a diagnosis of acute myeloid leukemia (AML) with a specific genetic mutation known as FLT3-ITD.

The patient is between 1 month and 21 years old and has met all the necessary health criteria to participate.

2 re-induction cycle 1

The patient begins the first cycle of treatment, known as Re-Induction Cycle 1.

During this cycle, the patient receives a combination of chemotherapy drugs and quizartinib, an oral solution taken by mouth.

The goal is to achieve a state called complete remission (CR) or CR with incomplete recovery (CRi).

3 re-induction cycle 2

If necessary, the patient may undergo a second cycle of re-induction treatment.

The same combination of chemotherapy and quizartinib is administered to further reduce cancer cells.

4 optional consolidation therapy

Following the re-induction cycles, the patient may receive additional chemotherapy, known as consolidation therapy, to help maintain remission.

5 single-agent continuation therapy

The patient transitions to a phase where only quizartinib is administered.

This phase can last up to 12 cycles, aiming to sustain remission and prevent relapse.

6 monitoring and follow-up

Throughout the trial, the patient’s health and response to treatment are closely monitored.

Regular assessments are conducted to evaluate the effectiveness and safety of the treatment.

Who Can Join the Study?

  • The patient must have a diagnosis of Acute Myeloid Leukemia (AML), which is a type of blood cancer, with at least 5% of abnormal cells (blasts) in the bone marrow.
  • Male patients must be surgically sterile or agree to use highly effective birth control during treatment and for 6 months after the last dose of the study drugs.
  • The patient or their legal representative must understand the study’s experimental nature, potential risks, and benefits, and sign a written consent form.
  • Female patients must not donate or retrieve eggs for their own use from the time of screening and throughout the treatment period, and for at least 6 months after the last dose.
  • Male patients must not freeze or donate sperm starting at screening and throughout the treatment period, and for at least 6 months after the last dose.
  • The patient must be in their first relapse or not responding to the first round of high-dose chemotherapy, with no more than one attempt at remission induction. Previous Hematopoietic Stem Cell Transplant (HSCT) is allowed.
  • The patient must have a specific genetic mutation called FLT3-ITD in their bone marrow or blood.
  • The patient must be between 1 month and 21 years old at the time of signing the consent form.
  • The patient must have a performance status score that meets the study’s requirements, indicating they are well enough to participate.
  • The patient must have fully recovered from any significant side effects of previous cancer treatments before starting the new treatment cycle.
  • The patient must have adequate kidney, liver, and heart function as defined by the study.
  • If the patient can have children, they must be permanently sterile or agree to use highly effective birth control during the study and for 6 months after the last dose of the study drugs.
  • If the patient is a female who can become pregnant, she must test negative for pregnancy and agree not to breastfeed during the study.

Who Cannot Join the Study?

  • Patients who have a different type of cancer other than Acute Myeloid Leukemia cannot participate. Acute Myeloid Leukemia is a type of blood cancer.
  • Patients who are not within the specified age range of 1 month to 21 years old are excluded.
  • Patients who are not able to safely receive the study medication or chemotherapy due to other health conditions are excluded.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may be excluded.
  • Patients who have certain infections or other serious health issues that could interfere with the study are excluded.
  • Patients who are unable to follow the study procedures or attend required visits are excluded.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name City Country Status
Hopital Des Enfants Toulouse France
Universitair Ziekenhuis Gent Gent Belgium
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Fondazione IRCCS San Gerardo Dei Tintori Monza Italy
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Prinses Maxima Centrum voor Kinderoncologie B.V. Utrecht The Netherlands
Assistance Publique Hopitaux De Paris Paris France
Rigshospitalet Copenhagen Denmark
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Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
15.08.2018
Denmark Denmark
Not recruiting
15.08.2018
France France
Not recruiting
15.08.2018
Italy Italy
Not recruiting
15.08.2018
Spain Spain
Not recruiting
15.08.2018
Sweden Sweden
Not recruiting
15.08.2018
The Netherlands The Netherlands
Not recruiting
15.08.2018

Trial locations

Investigated drugs:

Quizartinib is a medication being studied for its potential use in treating pediatric patients with a specific type of leukemia known as acute myeloid leukemia (AML) that has returned or is resistant to treatment. This medication is being tested in combination with chemotherapy to see if it can help improve the effectiveness of the treatment. Quizartinib is also being evaluated as a standalone therapy to see if it can help maintain remission in patients after they have completed chemotherapy. The study aims to find the best dose of quizartinib for different age groups and to understand how the body processes the medication.

Acute Myeloid Leukemia – Acute Myeloid Leukemia (AML) is a type of cancer that starts in the bone marrow, where blood cells are made, and quickly moves into the blood. It affects a group of white blood cells called myeloid cells, which normally develop into various types of mature blood cells. In AML, these cells do not mature properly and multiply rapidly, leading to a buildup of abnormal cells that can crowd out normal blood cells. This can result in symptoms such as fatigue, frequent infections, and easy bruising or bleeding. The disease can progress quickly, requiring prompt medical attention. AML is more common in adults but can also occur in children.

Trial ID:
2023-510009-16-00
Protocol code:
AC220-A-U202
NCT ID:
NCT03793478
Trial Phase:
Human Pharmacology (Phase I) – Other

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