Study Comparing Modified LCH-III Chemotherapy With or Without Luvometinib for Children With Multisystem Langerhans Cell Histiocytosis

What this Trial Is About

Langerhans cell histiocytosis LCH is a disorder mostly affecting children, caused by an abnormal buildup of Langerhans cells in body tissues. This disease varies widely in severity, with mild cases often resolving on their own and severe multisystem cases involving multiple organs like liver, spleen, or bone marrow, which can be life-threatening without proper treatment. Standard treatment includes prednisone and vinblastine chemotherapy, but some children develop resistance or relapse, leading to long-term health problems. This trial studies whether adding a targeted drug called luvometinib to chemotherapy can improve outcomes in children with multisystem LCH. Participants will be randomly assigned to receive either a modified standard chemotherapy regimen prednisone and vincristine alone or the same chemotherapy combined with oral luvometinib, a MEK12 inhibitor taken daily. The chemotherapy also includes mercaptopurine during maintenance. The study is conducted at multiple centers and aims to evaluate if the combination treatment improves patient outcomes compared to chemotherapy alone. During the trial, children will be closely monitored through clinical and imaging evaluations. Researchers will assess event-free survival over two years, responses at 1 and 3 months after treatment, overall survival, and any treatment-related side effects. Follow-up will include safety assessments and regular check-ups to track disease progression and treatment tolerance throughout the study period.

Modified LCH-III Regimen With or Without Luvometinib for Multisystem Pediatric Langerhans Cell Histiocytosis