Langerhans cell histiocytosis (LCH) is a rare disease where the body produces too many immature Langerhans cells, a type of white blood cell. These cells can build up and form tumors in organs like bones, skin, lungs, and the pituitary gland, causing damage. This condition is more common in children and young adults.
Tovorafenib is a medication that may help by blocking enzymes needed for cell growth, potentially stopping the spread of these abnormal cells. This study is important because it focuses on patients whose disease has returned or does not respond to standard treatments. Researchers will also analyze biomarkers and genetic mutations to understand how the treatment works and who might benefit most.
Eligibility
Age between 180 days and less than 22 years
Diagnosis of progressive, relapsed, or refractory LCH with measurable disease
Histologic confirmation of LCH from original diagnosis or relapse
