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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
40
Mirdametinib is administered by mouth twice daily on a continuous schedule, with each cycle being 4 weeks. Patients are instructed to take consecutive doses separated by a minimum of 6 hours and a maximum of 14 hours.
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
RECRUITINGResponse rate to mirdametinib
Best overall response rate to mirdametinib after 13 four-week cycles as defined by positron emission tomography (PET) or magnetic resonance imaging (MRI) (for isolated pituitary/central nervous system (CNS) disease) response criteria.
Time frame: 1 year (completion of 13 four week cycles)
Duration of response to mirdametinib
Duration of response to mirdametinib on study as defined by PET or MRI (for isolated pituitary/CNS disease) response criteria.
Time frame: 2 years (completion of 26 four week cycles)
Maximum Plasma Concentration (Cmax)
Blood samples will be drawn for the pharmacokinetic profile before, and during mirdametinib therapy. Maximum plasma concentration will be calculated as data allow.
Time frame: Day 1 of the first 5 four week cycles)
Time to peak drug concentration (Tmax)
Blood samples will be drawn for the pharmacokinetic profile before, and during mirdametinib therapy. Time to peak drug concentration (Tmax) will be calculated as data allow.
Time frame: Day 1 of the first 5 four week cycles)
Area under the plasma concentration time curve (AUC)
Blood samples will be drawn for the pharmacokinetic profile before, and during mirdametinib therapy. Area under the plasma concentration time curve (AUC) will be calculated as data allow.
Time frame: Day 1 of the first 5 four week cycles)
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