Study on the Dose and Safety of Asciminib in Children with Chronic Myeloid Leukemia

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What is this study about?

This clinical trial is focused on studying a type of blood cancer called Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) in children. This condition is a form of leukemia, which is a cancer of the blood and bone marrow. The study is testing a medication called Asciminib, also known by its code name ABL001. Asciminib is taken as a film-coated tablet and is designed to be used by patients who have already been treated with other medications known as tyrosine kinase inhibitors.

The purpose of the study is to determine the appropriate dose and safety of Asciminib in children with this type of leukemia. The study will involve giving the medication to pediatric patients and monitoring how their bodies process the drug. This will help researchers understand how the drug behaves in children compared to adults. The study will also look at the safety of the medication by observing any side effects that may occur during the treatment period.

Participants in the study will take Asciminib orally, and the study will track their response to the medication over time. The researchers will collect information on how well the drug is tolerated and its effects on the leukemia. The study aims to find a dose that provides similar drug exposure in children as seen in adults, ensuring the treatment is both effective and safe for younger patients. The study will also gather feedback on how acceptable and palatable the medication is for the children taking it.

1 joining the study

Upon joining the study, the participant will be informed about the trial’s purpose and procedures. The study focuses on determining the dose and safety of asciminib in pediatric patients with a specific type of leukemia.

Participants must be pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase, previously treated with one or more tyrosine kinase inhibitors.

2 medication administration

Participants will receive asciminib, which is administered orally in the form of film-coated tablets.

The goal is to achieve a dosage that provides similar exposure to 40 mg taken twice daily by adults.

3 monitoring and assessments

Throughout the study, participants will undergo regular monitoring to assess the pharmacokinetic profile of asciminib. This includes measuring how the drug is absorbed, distributed, metabolized, and excreted by the body.

Participants will also be evaluated for any side effects or adverse events related to the treatment.

4 response evaluation

The study will assess the hematologic and molecular responses to the treatment. This involves checking blood and bone marrow for changes in leukemia cells.

Participants will complete a questionnaire on the acceptability and taste of the medication after the first dose, and again at 4 and 52 weeks.

5 safety and growth assessments

Safety data will be collected, including assessments of growth and sexual maturation, to ensure the treatment does not adversely affect development.

The study will continue until the estimated end date in June 2031, with ongoing evaluations to ensure participant safety and treatment efficacy.

Who Can Join the Study?

The study is for both male and female participants.
Participants must be 1 to less than 18 years old for the pediatric group.
For the adult formulation group, participants must be 14 to less than 18 years old and weigh at least 40 kg (about 88 pounds).
Participants must have a specific type of leukemia called Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).
Participants must have been treated with at least one tyrosine kinase inhibitor (TKI) before. TKIs are a type of medication used to treat certain cancers.
Participants must have had a failure or intolerance to their most recent TKI therapy.
Participants must have a specific genetic marker called the BCR-ABL fusion gene, which can be measured with a test called real-time quantitative polymerase chain reaction (RQ-PCR).
Participants must meet certain blood test requirements at the screening visit, including:
- Less than 15% blasts in blood and bone marrow.
- Less than 30% combined blasts and promyelocytes in blood and bone marrow.
- Less than 20% basophils in the blood.
- Neutrophils (a type of white blood cell) must be at least 1.5 x 10⁹/L, or if not available, white blood cells must be at least 3 x 10⁹/L.
- Platelet count must be at least 100 x 10⁹/L.
- No signs of leukemia outside the bone marrow, except for enlarged liver or spleen (hepatosplenomegaly).

Who Cannot Join the Study?

Patients who have not been diagnosed with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). This is a type of blood cancer that has a specific genetic marker called the Philadelphia chromosome.
Patients who have not been previously treated with one or more tyrosine kinase inhibitors. These are medications used to treat certain types of cancer by blocking signals that cause cancer cells to grow.
Patients who are not within the specified age range for the study. The study is for pediatric patients, which means children.
Patients who are not able to take the study medication as required, such as those who cannot swallow pills or have dietary restrictions that prevent them from taking the medication with food.
Patients who have other medical conditions or are taking other medications that might interfere with the study treatment or affect their safety during the study.
Patients who are pregnant or breastfeeding, as the study medication may not be safe for unborn babies or infants.
Patients who are unable to comply with the study procedures, such as attending regular visits or following the study instructions.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Uniwersytecki Szpital Kliniczny Im Jana Mikulicza Radeckiego We Wroclawiu	Wroclaw	Poland
Centre Hospitalier Universitaire De Lille	Lille	France

Other Sites

Site Name	City	Country
Centre Hospitalier Universitaire De Poitiers	Poitiers	France
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Nosokomeio Paidon I Agia Sofia	Athens	Greece
Fondazione IRCCS San Gerardo Dei Tintori	Monza	Italy
Universitaetsklinikum Erlangen AöR	Erlangen	Germany
Semmelweis University	Budapest	Hungary
Robert Debre University Hospital	Paris	France
Prinses Maxima Centrum voor Kinderoncologie B.V.	Utrecht	The Netherlands
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Uogaqqmdwo Mgosudt Cshcgn Hqdcxhevqmjkddxhk	Hamburg	Germany
Uolxmbmjfdqmdlpuqxepi Eyxop Ato	Essen	Germany
Aglzvvl Ouctdjhevgc Ublroswrgsxmt Cgqrlynflwxj Dtcmk Stlufs E Dbswc Sbjrrwr Dn Tbohvt	Turin	Italy

Trial status

Country	Status	Recruitment Start
France	Recruiting	28.09.2022
Germany	Recruiting	28.09.2022
Greece	Recruiting	28.09.2022
Hungary	Not recruiting	28.09.2022
Italy	Recruiting	28.09.2022
Poland	Recruiting	28.09.2022
The Netherlands	Recruiting	28.09.2022

Trial locations

Investigated drugs:

Asciminib is a medication being studied for its use in treating pediatric patients with a specific type of leukemia known as Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). This medication has been previously used in adults and is now being tested to find the right dose for children. The study aims to understand how the body processes asciminib in children to ensure it is safe and effective for them.

Chronic Myeloid Leukemia in Chronic Phase (Ph+ CML-CP) – This is a type of cancer that affects the blood and bone marrow, characterized by the presence of the Philadelphia chromosome. It primarily involves the overproduction of white blood cells. In the chronic phase, the disease progresses slowly, and patients may not exhibit significant symptoms initially. Over time, the number of abnormal cells increases, potentially leading to symptoms such as fatigue, weight loss, and an enlarged spleen. The chronic phase can last for several years before potentially advancing to more aggressive phases. This condition is often managed with medications that target specific proteins involved in the growth of cancer cells.

Trial ID:

2023-508129-28-00

Protocol code:

CABL001I12201

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on the Dose and Safety of Asciminib in Children with Chronic Myeloid Leukemia

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