Philadelphia chromosome positive Trials in Disease

Study of dasatinib treatment in children and adolescents with Philadelphia chromosome-positive leukemia who cannot take imatinib or for whom imatinib is not effective

This clinical trial is designed for children and adolescents with Chronic Myeloid Leukemia (CML) and Philadelphia chromosome-positive leukemias who have either been newly diagnosed or have not responded well to imatinib treatment. The study tests dasatinib, also known by its brand name Sprycel, which is available as tablets or as a powder that can be mixed into a liquid for easier administration.

Main inclusion criteria:

• Age between 1 and 18 years, both males and females
• Written informed consent must be provided
• Patients must fall into one of three groups: those with chronic phase CML who did not respond to or cannot tolerate imatinib, those with Ph+ ALL or advanced phase CML who did not respond to or cannot tolerate imatinib, or those newly diagnosed with chronic phase CML
• Ability to perform daily activities must be above 50 on the Lansky or Karnofsky score (scales that measure functional ability)
• Life expectancy of at least 12 weeks
• Blood tests must show adequate levels of important minerals, and liver and kidney function must be sufficient
• Patients must have recovered from side effects of previous treatments to normal or mild levels

Main exclusion criteria:

• Previous treatment with any BCR-ABL inhibitor other than imatinib
• Presence of the T315I mutation, a genetic change that makes cancer resistant to certain treatments
• Active involvement of the central nervous system by leukemia
• Significant heart problems, including uncontrolled heart disease, recent heart attack, or unstable heart rhythm disorders
• Severe liver or kidney problems
• Active uncontrolled infections
• Pregnancy or breastfeeding
• Inability to take oral medications
• Known allergic reaction to dasatinib or similar medications

Focus and goal: The primary purpose of this trial is to evaluate how well dasatinib works in young patients whose leukemia has either become resistant to imatinib or who cannot tolerate imatinib's side effects. The study will also assess dasatinib's effectiveness in newly diagnosed patients with chronic phase CML. Doctors will monitor several important measures of treatment success, including how well the medication controls blood cell counts and whether it reduces or eliminates cancer cells containing the Philadelphia chromosome. Throughout the study, participants will undergo regular blood count checks, bone marrow examinations, and monitoring for side effects. The research team will measure treatment response by tracking whether blood cell counts return to normal levels and whether there is a reduction in abnormal cells in the bone marrow.

Investigational drug: The study uses dasatinib, a medication that blocks specific proteins that cause cancer cells to grow and divide. This targeted therapy is specifically designed for patients whose cancer has become resistant to other treatments or who cannot tolerate them. Dasatinib belongs to a class of medications called tyrosine kinase inhibitors. The trial compares dasatinib to imatinib, which was the previous standard treatment and works in a similar way by targeting specific proteins in cancer cells. Dasatinib is being studied as an alternative for patients who did not respond well to imatinib or couldn't tolerate its side effects.