Study on the Safety and Effectiveness of Quizartinib for Children with Newly Diagnosed FLT3-ITD Positive AML and Normal NPM1

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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) in children and adolescents. Specifically, it looks at cases where the cancer cells have a particular genetic change known as FLT3-ITD and do not have another change called NPM1 mutation. The study is testing a treatment using a medication called Quizartinib, which is taken as an oral solution. Quizartinib is being tested both in combination with standard chemotherapy and on its own after high-dose therapy.

The purpose of the study is to evaluate the safety and effectiveness of Quizartinib in treating this specific type of AML. Participants will receive up to two courses of chemotherapy along with Quizartinib. The study will monitor how well the treatment works by checking if the cancer cells are reduced to very low levels in the bone marrow. This is done using a method called flow cytometry, which helps in measuring the number of cancer cells. The study will also assess the safety of Quizartinib by observing any side effects that occur during the treatment period.

Throughout the study, participants will be evaluated at different stages to see how their bodies respond to the treatment. This includes checking for any adverse effects and measuring the levels of cancer cells in the bone marrow. The study aims to find the most suitable dose of Quizartinib that can be safely given to children and adolescents with this type of AML. The overall goal is to improve treatment outcomes for young patients with this challenging form of leukemia.

1 joining the study

Upon joining the study, you will be required to provide written informed consent or assent. This is a formal agreement to participate, which must be signed by you or your parent/legal guardian if you are a minor.

2 initial assessment

An initial assessment will be conducted to confirm eligibility. This includes checking your age, health status, and specific medical conditions. You must be between 1 month and 18 years old, with a specific type of leukemia known as FLT3-ITD positive and NPM1 wild-type AML.

3 medication administration

You will receive a medication called Quizartinib, which is an oral solution. This means you will take it by mouth. The dosage and frequency will be determined by the study team based on your specific needs and the study protocol.

4 chemotherapy combination

Quizartinib will be administered in combination with chemotherapy. This phase is designed to assess the safety and effectiveness of the treatment. The study will monitor your response to the treatment and any side effects.

5 monitoring and evaluation

Throughout the study, regular evaluations will be conducted. These include checking your bone marrow to measure the level of leukemia cells and assess the treatment’s effectiveness. This is done using a technique called flow cytometry.

6 safety assessment

The study will also focus on safety by monitoring any side effects or adverse reactions to the treatment. This is crucial to determine the recommended dose for future patients.

7 completion of study

Upon completion of the study, a final assessment will be conducted to evaluate the overall effectiveness and safety of the treatment. This will help in understanding the long-term benefits and risks associated with the medication.

Who Can Join the Study?

Must be enrolled in the CHIP-AML22/Master study.
Must have a specific type of leukemia called FLT3-ITD positive and NPM1 wild-type.
Age must be between 1 month and 18 years old at the time of initial diagnosis.
For those older than 16 years, a Karnofsky Performance status of more than 50% is required. For those 16 years or younger, a Lansky performance status score of more than 50% is needed. These scores measure the ability to perform daily activities.
Must have adequate organ function:
- Renal (kidney) function: A calculated eGFR (a test of kidney function) of at least 50 mL/min/1.73 m².
- Liver function: Total or direct bilirubin (a liver function test) less than 1.5 times the upper limit of normal for age, or less than 5 times if related to leukemia. AST and ALT (liver enzymes) less than 5 times the upper limit of normal, or less than 10 times if related to leukemia.
Must have a life expectancy of more than 6 weeks.
If applicable, a negative pregnancy test is required within 2 weeks before joining the quizartinib trial.
Must be able to swallow or take the medication quizartinib through a nasogastric (NG) tube, which is a tube that goes through the nose to the stomach.
Must provide written informed consent or assent for the quizartinib trial. For minors, consent must be given by parents or legal guardians according to local laws and regulations.

Who Cannot Join the Study?

Patients who do not have a newly diagnosed condition called FLT3-ITD positive and NPM1 wild-type AML cannot participate. This is a specific type of acute myeloid leukemia (AML) found in children.
Patients who have a different type of genetic mutation in their AML, other than FLT3-ITD positive and NPM1 wild-type, are not eligible.
Patients who are not within the specified age range for the study cannot participate. The study is for children.
Patients who are not able to safely tolerate the study medication, quizartinib, based on their health condition, are excluded.
Patients who have any other medical conditions that might interfere with the study treatment or its evaluation are not eligible.
Patients who are part of a vulnerable population that cannot safely participate in the study are excluded.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Oslo Universitetssykehus HF	Oslo	Norway
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Katholieke Universiteit te Leuven	Leuven	Belgium

Other Sites

Site Name	City	Country
Universitetssykehuset Nord-Norge HF	Tromsø	Norway
Prinses Maxima Centrum voor Kinderoncologie B.V.	Utrecht	The Netherlands
Region Oestergoetland	Linkoping	Sweden
Region Vaesterbotten	Umea	Sweden
Universitair Ziekenhuis Gent	Gent	Belgium
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Centre Hospitalier Regional De La Citadelle	Liege	Belgium
Hospital General Universitario Gregorio Maranon	Madrid	Spain
St. Olavs Hospital HF	Trondheim	Norway
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
Instituto Portugues De Oncologia Do Porto Francisco Gentil E.P.E.	Porto	Portugal
Oulu University Hospital	Oulu	Finland
Complejo Hospitalario Universitario Insular Materno Infantil	Las Palmas De Gran Canaria	Spain
Virgen del Rocío University Hospital	Sevilla	Spain
Region Skane Skanes Universitetssjukhus	Lund	Sweden
Vilniaus universiteto ligonine Santaros klinikos VšĮ	Vilnius	Lithuania
Tartu University Hospital	Tartu	Estonia
Karolinska University Hospital	Solna	Sweden
Hospital Universitario De Cruces	Barakaldo	Spain
Instituto Portugues De Oncologia De Lisboa Francisco Gentil E.P.E.	Lisbon	Portugal
Turku University Hospital	Turku	Finland
University Clinical Hospital Virgen De La Arrixaca	Murcia	Spain
Universidade De Santiago De Compostela	Santiago De Compostela	Spain
CHC MontLegia	Liege	Belgium
Rigshospitalet	Copenhagen	Denmark
Pohjois-Savon hyvinvointialue	Kuopio	Finland
Pirkanmaan hyvinvointialue	Tampere	Finland
University Childrens Hospital Queen Fabiola	Brussels	Belgium
Servei De Salut De Les Illes Balears	Palma	Spain
Tallinn Children´s Hospital	Tallin	Estonia
Cgjfwibor Udvijoxgwgeana Scwfsjehi	Woluwe-Saint-Lambert	Belgium
Hlamfhtq Ufbtyeyneuvpl Mimxbji Ds Vekdkwauvm	Santander	Spain
Bhqic Kyzsyvgs Ugsypdzfkdfar Suqlikhw Vigc	Riga	Latvia
Ubdqvum Uarvmeelsz Hjgyyasi	Uppsala	Sweden
Hkqkz Boxjbb Hq	Bergen	Norway
Hxxhxdke Ukobcgbqnv Cttkffa Hclemjrv	Helsinki	Finland
Aonahi Uqbyuuguor Hhmxtuou	Aarhus	Denmark
Ftceuevzu Pzng Lr Iipriuhkvshfx Baxehwgbv Dsa Hmdtjifx Ulqrcnzfweazi Lu Pvr	Madrid	Spain
Hqppndao Vwak dxbiysox	Barcelona	Spain
Cxhdww Hvxxzpadfj E Uatlxaecbdkgw Dd Ckptvob Egectz	Coimbra	Portugal

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	31.12.2023
Denmark	Recruiting	31.12.2023
Estonia	Recruiting	31.12.2023
Finland	Recruiting	31.12.2023
Iceland	Recruiting	31.12.2023
Latvia	Recruiting	31.12.2023
Lithuania	Recruiting	31.12.2023
Norway	Recruiting	31.12.2023
Portugal	Not yet recruiting	31.12.2023
Spain	Recruiting	31.12.2023
Sweden	Recruiting	31.12.2023
The Netherlands	Recruiting	31.12.2023

Trial locations

Investigated drugs:

Quizartinib Dihydrochloride

Quizartinib is a medication being studied for its potential to help treat a type of blood cancer called acute myeloid leukemia (AML) in children. This medication is being tested to see if it can work well with chemotherapy and also on its own after high-dose therapy. The goal is to see if it can help reduce the number of cancer cells in the body, especially in children who have a specific genetic marker called FLT3-ITD and do not have another marker called NPM1 mutation. Researchers are also looking at how safe quizartinib is for children and what the best dose might be to use in future treatments.

Investigated diseases:

Acute myeloid leukaemia

Acute Myeloid Leukemia (AML) with FLT3-ITD Mutation and NPM1 Wild-Type – Acute Myeloid Leukemia (AML) is a type of cancer that affects the blood and bone marrow, characterized by the rapid growth of abnormal white blood cells. In this specific form, there is a mutation in the FLT3 gene known as FLT3-ITD, which leads to uncontrolled cell division. The NPM1 gene remains in its normal, wild-type state, which can influence the disease’s behavior and response to treatment. The disease progresses as these abnormal cells accumulate, interfering with the production of normal blood cells. This can lead to symptoms such as fatigue, frequent infections, and easy bruising or bleeding. Over time, the disease can advance rapidly, requiring prompt medical attention.

Trial ID:

2023-505000-27-01

Protocol code:

MH22CAQ

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Safety and Effectiveness of Quizartinib for Children with Newly Diagnosed FLT3-ITD Positive AML and Normal NPM1

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?