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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 is an oral medication that 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. DAY101 is considered investigational, which means that it is not yet approved by the U.S. Food and Drug Administration (FDA) for this purpose.
Participants will take DAY101 once a week for cycles of 28 days and the study team will discuss dose levels with each participant.
Up to 12 participants will first be enrolled and observed for safety of DAY1010. If there are less than 2 dose limiting toxicities during the first 2 cycles of therapy, these will be considered the recommended phase II dose, and all subsequent participants will be enrolled at these doses. If participants tolerate the recommended phase II dose, then the Phase 2 study cohort will be initiated. Patients for the Phase 2 study may be included for analysis at the recommended phase II dose from the dose-finding cohort.
Inclusion Criteria:
1. Male and female patients younger than 22 years old
2. Patients with multifocal progressive, relapsed, or recurrent Langerhans Cell Histiocytosis with measurable disease at study entry.
3. Patients with mixed histiocytic disorders (e.g., Langerhans Cell Histiocytosis (LCH) with juvenile xanthogranuloma) may be included.
4. Participant must be able to take an enteral dose and formulation of medication. Study medication is only available as an oral suspension or tablet, which may be taken by mouth or other enteral route such as nasogastric, jejunostomy, or gastric tube.
Exclusion Criteria:
1. Female patients who are pregnant are ineligible. A pregnancy test is required for female patients of childbearing potential.
2. Patient must not have received any prior MAPK pathway inhibitor therapy.
3. Patients with activating mutations in MAP2K1 are not eligible for this study due to drug target specificity. Mutation status will be submitted to study team within 7 days of enrollment.
Detailed eligibility will be reviewed when you contact the study team.
Upper East Side - Manhattan
Not Yet Recruiting
