Histiocytic Disorders Clinical Trial
Official title:
A Phase II Trial of the MEK Inhibitor Mirdametinib in Histiocytic Disorders
The purpose of this study is to see if treatment with mirdametinib in patients with Langerhans cell histiocytosis (LCH) or other histiocytic disorders will be better than current treatments and with fewer side effects.
Langerhans cell histiocytosis (LCH) is a rare blood disorder. Though affecting all ages, LCH occurs more often in children, with an increased incidence in children less than 1 year of age. The disease presents in various ways, with most children suffering bony lesions, and skin rashes. In some patients, LCH affects vital organs such as liver, spleen, bone marrow, and the central nervous system. This group of patients are at significant risk of serious illness and death and are thus said to have risk-organ-positive (RO+) LCH. Current treatments for LCH consist of chemotherapy combined with other medications. However, many patients, especially those with RO+ disease, do not respond to therapy. Of the patients that do respond, many suffer progression of disease after an initial response to therapy, or recurrence of disease after completion of therapy. The purpose of this study is to see if treatment with mirdametinib in patients with LCH or other histiocytic disorders will be better than current treatments and with fewer side effects. ;
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