Study on the Safety and Effectiveness of Bezuclastinib for Patients with Advanced Systemic Mastocytosis
This international clinical trial is investigating Bezuclastinib, also known as CGT9486, as a potential treatment for patients with advanced forms of mastocytosis. The condition involves the uncontrolled growth and accumulation of mast cells in various organs throughout the body, which can cause a range of symptoms and organ dysfunction.
Who can participate: This study is open to patients who have been diagnosed with one of three specific types of advanced mastocytosis: Aggressive Systemic Mastocytosis, Systemic Mastocytosis with an Associated Hematologic Neoplasm, or Mast Cell Leukemia. Participants must have measurable disease and an ECOG performance status between 0 and 3, which is a scale doctors use to assess how well a person can perform daily activities. A score of 0 means the person is fully active, while 3 means they have limited ability to care for themselves. Blood tests and other laboratory results must fall within clinically acceptable ranges. Both male and female patients can participate, and the study includes vulnerable populations who may need special consideration or protection.
Who cannot participate: Patients who do not have Advanced Systemic Mastocytosis are excluded from the study. Additionally, only patients within a specified age range can join, and those who do not meet other specific clinical criteria or gender-specific requirements cannot participate.
What the study involves: The trial is structured in multiple parts. In the first phase, called dose optimization, researchers will work to identify a safe and effective dose of Bezuclastinib. Participants will take the medication as an oral tablet, and doctors will monitor their response and any side effects. Once the optimal dose is identified, the study moves to a dose confirmation stage, where this dose is tested further for safety and effectiveness. The final expansion stage aims to determine how well the medication works at the confirmed dose. Throughout the entire trial, researchers will monitor various health parameters including blood and bone marrow markers, particularly focusing on the KIT D816V mutation and serum tryptase levels, which are commonly involved in this disease.
Investigational drug: Bezuclastinib is a type of medication called a tyrosine kinase inhibitor. It works at the molecular level by blocking specific enzymes involved in the growth and survival of mast cells. The drug targets mutations in a protein called KIT kinase, which often plays a role in the uncontrolled growth of mast cells in this condition. Bezuclastinib is taken orally as a tablet and is still under investigation, meaning it is not yet widely available in standard medical practice.
The study is expected to continue until 2027, providing valuable information about the potential benefits and risks of this treatment approach. Researchers will also assess the impact of the medication on patients' quality of life, symptom changes, progression-free survival, and overall survival.
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