Phase II Study of Oral Tovorafenib DAY101 for Children and Young Adults with Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis

What this Trial Is About

Researchers are evaluating tovorafenib DAY101 in a phase II trial for children and young adults with progressive, relapsed, or refractory Langerhans cell histiocytosis LCH. This disease involves an overproduction of immature Langerhans cells that form tumors in tissues like bones, skin, lungs, and the pituitary gland. The trial aims to determine the overall response rate, side effects, event-free survival, and durability of response to this oral treatment, focusing on patients with specific genetic mutations. Participants receive tovorafenib orally once a week on days 1, 8, 15, and 22 of each 28-day cycle, for up to 12 cycles unless the disease progresses or side effects become unacceptable. Throughout the study, patients undergo various scans including MUGA or echocardiography, FDG-PET or CT, and provide blood and urine samples. Those suspected of bone marrow or central nervous system involvement will have additional procedures like bone marrow biopsy, aspiration, and lumbar puncture. During the trial, researchers assess patient response using imaging and laboratory tests, monitor adverse events, and collect samples to study genetic mutations and drug effects. After treatment ends, participants have follow-up visits at 28 days, and at 3, 6, 9, 12 months, and two years post-treatment to evaluate long-term outcomes and safety. The study measures include overall response after two cycles and survival rates up to two years.

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