Ongoing Clinical Trials for Philadelphia Positive Chronic Myeloid Leukaemia
Currently, there are 3 ongoing clinical trials investigating new treatment approaches for Philadelphia positive chronic myeloid leukaemia. These studies are being conducted across several European countries and focus on different tyrosine kinase inhibitors, including asciminib, ponatinib, and nilotinib. The trials aim to improve treatment outcomes, explore dosing in children, and investigate the potential for treatment-free remission in patients who have achieved deep molecular responses.
Clinical trial locations
- France
- Germany
- Greece
- Hungary
- Italy
- Netherlands
- Poland
- Portugal
Study on the Dose and Safety of Asciminib in Children with Chronic Myeloid Leukemia
This trial is investigating the appropriate dose and safety profile of asciminib in children with the chronic phase of this condition. Asciminib is a tyrosine kinase inhibitor that works through a unique mechanism by targeting the ABL myristoyl pocket, helping to block the growth of cancer cells.
Who can participate: The study is open to children and adolescents aged between 1 and 17 years who have been previously treated with one or more tyrosine kinase inhibitors and have experienced treatment failure or intolerance. Participants must have specific genetic markers including the BCR-ABL fusion gene and meet certain blood test requirements, such as having less than 15% blasts in blood and bone marrow, adequate neutrophil or white blood cell counts, and sufficient platelet levels.
Who cannot participate: Children who have not been diagnosed with this specific form of chronic myeloid leukemia, those who have not received prior tyrosine kinase inhibitor treatment, patients unable to take the medication as required, those with medical conditions that might interfere with treatment, and pregnant or breastfeeding patients are excluded from the study.
Study focus: The research aims to determine a dosage for children that provides similar drug exposure as seen in adults taking 40 mg twice daily. Researchers will monitor how children’s bodies process the medication, assess safety by observing side effects, and evaluate both blood and bone marrow responses to treatment. The study will also collect information on the acceptability and taste of the medication for young patients and include assessments of growth and sexual maturation to ensure treatment does not adversely affect development.
The trial is being conducted across multiple European countries including Italy, France, Germany, Hungary, Poland, the Netherlands, and Greece, with an estimated end date in June 2031.
Study on Ponatinib for Achieving Treatment-Free Remission in Patients with Chronic Myeloid Leukemia
This German trial is investigating whether patients who achieve a deep molecular response called MR4 after two years of treatment with ponatinib can potentially stop treatment while maintaining their response. Ponatinib is a tyrosine kinase inhibitor that blocks proteins helping cancer cells grow.
Who can participate: Adults aged 18 years or older with chronic phase disease who have the Philadelphia chromosome or BCR-ABL fusion gene. Participants must have a BCR-ABL IS level between 0.5 and 0.01, must not have achieved MR4 or stable MR4 after at least 3 years of treatment with medications like nilotinib, dasatinib, or bosutinib, and must have had an eye examination within 8 weeks before starting treatment.
Who cannot participate: Patients who have not achieved MR4 after two years of ponatinib treatment, those not in the chronic phase of the disease, patients outside the specified age range, and individuals considered vulnerable who might need special protection are excluded.
Study focus: Over 24 months, researchers will monitor participants’ molecular status at different response levels, assess the safety and tolerability of ponatinib, and evaluate quality of life. The study will identify clinical and biological factors associated with achieving MR4 or better responses, such as risk scores, gender, and duration of previous treatments. Long-term outcomes including overall survival and progression-free survival will be evaluated to understand the potential for treatment-free remission.
Study on the Bioavailability of Nilotinib in Healthy Individuals with Chronic Myelogenous Leukemia (CML)
This Portuguese trial is studying how the body absorbs and processes nilotinib, a tyrosine kinase inhibitor that blocks signals causing cancer cells to multiply. The study compares different forms of nilotinib capsules to ensure patients receive the correct amount of medication for it to be effective.
Who can participate: Healthy individuals aged 18 to 55 years with a body weight of at least 48 kg and a body mass index between 18.5 and 30.0. Participants must provide informed consent, be willing to follow study procedures including restrictions on alcohol and diet, and agree to use contraception as required. They must have no significant diseases in their medical history, no abnormalities in physical examination or heart tests, negative tests for HIV and hepatitis, and must be non-smokers or have not used tobacco products for at least 3 months.
Who cannot participate: Patients with primary Philadelphia chromosome positive leukemia cells from chronic myelogenous leukemia are not eligible for this particular bioavailability study.
Study focus: Participants will receive single doses of nilotinib 200 mg hard capsules in four different treatment periods with washout periods in between. The study uses a randomized, open-label design where participants receive medication in different sequences. Researchers will monitor health through ECG tests, vital signs, and laboratory tests to evaluate bioavailability, measuring how much of the drug is absorbed into the bloodstream and how long it stays in the body.
Summary
These three clinical trials represent diverse approaches to advancing treatment for Philadelphia positive chronic myeloid leukaemia across Europe. A notable observation is the geographical spread, with the pediatric asciminib trial being conducted in seven European countries, reflecting the challenges of recruiting sufficient numbers of children for rare disease studies. Germany appears twice in the trial locations, hosting both the ponatinib treatment-free remission study and participating in the pediatric asciminib research.
The trials address different aspects of disease management: one focuses specifically on the pediatric population, an often underserved group in clinical research; another investigates the possibility of stopping treatment in patients with excellent responses; and the third examines fundamental drug absorption properties. Each trial studies a different tyrosine kinase inhibitor—asciminib, ponatinib, and nilotinib—highlighting the continued development and refinement of this important drug class for treating this condition.
