Ongoing Clinical Trials for Advanced Systemic Mastocytosis
Currently, there is one clinical trial underway investigating treatment options for Advanced Systemic Mastocytosis, a rare disorder where mast cells accumulate uncontrollably in various organs. This international study is testing an investigational medication called Bezuclastinib across multiple European countries, focusing on finding a safe and effective dose for patients with this challenging condition.
Clinical trial locations
- Austria
- Belgium
- France
- Germany
- Italy
- Netherlands
- Norway
- Poland
- Spain
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.
Summary
This single ongoing clinical trial for Advanced Systemic Mastocytosis represents an important effort to develop new treatment options for this rare condition. The study has a notably broad geographic reach, spanning nine European countries including France, Austria, Spain, Norway, Germany, Netherlands, Italy, Belgium, and Poland, which may help ensure diverse patient participation and broader access to this investigational treatment.
The trial’s focus on Bezuclastinib, a tyrosine kinase inhibitor targeting KIT mutations, reflects current scientific understanding of the molecular mechanisms underlying this disease. The structured approach, moving from dose optimization through confirmation to expansion phases, demonstrates a careful methodology designed to ensure both safety and effectiveness are thoroughly evaluated before wider use.
For patients living with Advanced Systemic Mastocytosis, this trial may represent an opportunity to access a novel treatment approach while contributing to the advancement of medical knowledge about this challenging condition.