National Cancer Institute (NCI)
This phase II trial tests the safety, side effects, best dose and activity of tovorafenib (DAY101) in treating patients with Langerhans cell histiocytosis that is growing, spreading, or getting worse (progressive), has come back (relapsed) after previous treatment, or does not respond to therapy (refractory). Langerhans cell histiocytosis is a type of disease that occurs when the body makes too many immature Langerhans cells (a type of white blood cell). When these cells build up, they can form tumors in certain tissues and organs including bones, skin, lungs and pituitary gland and can damage them. This tumor is more common in children and young adults. DAY101 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Using DAY101 may be effective in treating patients with relapsed or refractory Langerhans cell histiocytosis.
PRIMARY OBJECTIVE: I. To determine overall response rate (ORR) for children and young adults with relapsed or refractory BRAFV600E positive (cohort 1) and BRAFV600E negative (cohort 2) Langerhans cell histiocytosis (LCH) treated with tovorafenib (DAY101) after 2 cycles and must be maintained 4 weeks later. SECONDARY OBJECTIVES: I. To determine nature and severity of adverse events in patients treated with tovorafenib (DAY101) for relapsed or refractory LCH. II. To describe event-free survival (EFS) at 1 year in children and young adults with relapsed and refractory LCH treated with tovoraf…
Inclusion Criteria: * 180 days- \< 22 years (at time of study enrollment) * Patient must have a body surface area of ≥ 0.3 m\^2 * Patients with progressive, relapsed, or recurrent LCH with measurable disease at study entry * Patients must have had histologic verification of LCH (from either original diagnosis or relapse/progression) at the time of study entry * Tissue confirmation of relapse is recommended but not required. * Pathology report must be submitted for central confirmation of diagnosis within 7 days of enrollment. * Formalin-fixed paraffin-embedded (FFPE) blocks or…
Undergo collection of blood and urine samples
Undergo bone marrow aspiration
Undergo bone marrow biopsy
Undergo CT
Undergo ECHO
Undergo FDG-PET imaging
Undergo lumbar puncture
Undergo MUGA
Given PO