A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refr
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). Langerhan
| Condition(s) | Recurrent Langerhans Cell Histiocytosis, Refractory Langerhans Cell Histiocytosis |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| Summary | 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 treat |
| Who can participate | 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 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 ju |
| Ages | 180 Days to 22 Years |
| Sex | All |
| Lead sponsor | National Cancer Institute (NCI) |
| Locations | Birmingham, Alabama, United States; Little Rock, Arkansas, United States; Downey, California, United States; Loma Linda, California, United States; Los Angeles, California, United States; Madera, California, United States (+111 more sites) |
| Start date | 2024-03-28 |
| NCT ID | NCT05828069 |
| Official listing | https://clinicaltrials.gov/study/NCT05828069 |