Ongoing Clinical Trials for Juvenile Chronic Myelomonocytic Leukaemia
There is currently 1 ongoing clinical trial investigating treatment options for patients with this blood cancer. The trial is evaluating a medication called Asciminib in children who have previously been treated with other cancer medications, and is being conducted across multiple European countries.
Clinical trial locations
- France
- Germany
- Greece
- Hungary
- Italy
- Netherlands
- Poland
Study on the Dose and Safety of Asciminib in Children with Chronic Myeloid Leukemia
This trial is investigating the appropriate dose and safety of Asciminib (also known as ABL001) in children with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase. The medication is taken as a tablet by mouth and has already been used in adults, but researchers need to determine the right dose for younger patients.
Main inclusion criteria:
- Children aged 1 to less than 18 years old (or 14 to less than 18 years old weighing at least 40 kg for the adult formulation group)
- Confirmed diagnosis of Philadelphia chromosome positive chronic myeloid leukemia in chronic phase
- Previous treatment with at least one tyrosine kinase inhibitor, a type of cancer medication
- The previous treatment must have failed or caused intolerable side effects
- Presence of a specific genetic marker called the BCR-ABL fusion gene
- Meeting specific blood test requirements, including limits on abnormal cells in blood and bone marrow, and adequate levels of certain blood cells
Main exclusion criteria:
- Patients without the specific type of leukemia being studied (Ph+ CML-CP)
- Patients who have never been treated with tyrosine kinase inhibitors
- Children outside the specified age range
- Patients unable to swallow pills or with dietary restrictions that prevent taking the medication with food
- Patients with other medical conditions or taking medications that might interfere with the study treatment
- Pregnant or breastfeeding patients
- Patients unable to attend regular visits or follow study instructions
Focus and goals: The trial aims to determine the correct dose of Asciminib that provides similar drug exposure in children as seen in adults taking 40 mg twice daily. Researchers will monitor how children’s bodies process the medication and observe any side effects that occur during treatment. The study will assess blood and bone marrow for changes in leukemia cells and evaluate the medication’s acceptability and taste from the children’s perspective. Safety assessments will include monitoring growth and sexual development to ensure the treatment does not negatively affect normal development. The study is expected to continue until June 2031.
Investigational drug: Asciminib works by specifically targeting a unique area called the ABL myristoyl pocket, which is a different mechanism compared to other tyrosine kinase inhibitors. This targeting approach helps to block the growth of cancer cells.
Summary
Currently, there is one active clinical trial for children with this specific type of blood cancer. The trial is notable for its broad European reach, being conducted across seven countries: Italy, France, Germany, Hungary, Poland, Netherlands, and Greece. This wide geographic distribution may help ensure diverse patient participation and data collection.
The trial focuses on Asciminib, a medication that has already been studied in adults and uses a unique mechanism to target cancer cells. The research is particularly important because it addresses the need to establish proper dosing and safety information specifically for pediatric patients, as children may process medications differently than adults.
The trial is designed as a long-term study, running until 2031, which will allow researchers to gather comprehensive information about both the immediate and long-term effects of the medication, including its impact on children’s growth and development.