RecruitingRecurrent Langerhans Cell HistiocytosisRefractory Langerhans Cell Histiocytosis

A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis

Eligible age

0–22 yrs

Accepts

All genders

Locations

38 states

Healthy volunteers

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About this study

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.

Sponsor: National Cancer Institute (NCI)

You may qualify if…

✓ 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 unstained slides (initial diagnosis and/or subsequent biopsies) will be required for retrospective central confirmation of diagnosis and molecular studies
✓ Patients with mixed histiocytic disorders (e.g. LCH with juvenile xanthogranuloma) may be included

You may not qualify if…

✕ LCH arising along with other hematologic malignancy (e.g. mixed LCH with acute lymphoblastic leukemia) or any history of non-histiocytic malignancy
✕ Disease scenarios as below will be excluded
✕ Skin-limited disease
✕ Gastrointestinal (GI) tract involvement only (those that have disease that can be determined by endoscopic biopsies only)
✕ LCH-associated neurodegeneration (LCH-ND) without parenchymal lesions or other systemic lesions
✕ 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
✕ Refractory nausea and vomiting, malabsorption, or external biliary shunt that would preclude adequate absorption of tovorafenib (DAY101)
✕ Uncontrolled systemic bacterial, viral, or fungal infection