This is a research study of a drug called cobimetinib in children and adults diagnosed with Langerhans cell histiocytosis (LCH), and other histiocytic disorders that has returned or does not respond to treatment. Cobimetinib blocks activation of a protein called Mitogen-activated protein kinase (MEK) that is part of incorrect growth signals in histiocytosis cells. Four different groups of patients will be enrolled.
Name: Cobimetinib
Description: Proportion of participants with (complete response, partial response, stable disease, progressive disease) by 1 year of therapy with Cobimetinib. It is assumed that at each protocol-specified timepoint, a response assessment occurs. Status calculation will occur at each timepoint for patients who have measurable disease at baseline per the criteria defined in the protocol.
Measure: Overall Response Rates using modified RECiST criteria Time: 12 monthsDescription: Progression Free Survival defined as the length of time during and after the treatment of a disease, that a patient lives with the disease but it does not get worse.
Measure: Progression Free Survival Time: 12 monthsDescription: Assessment of the nature and severity of adverse events in patients treated with Cobimetinib for histiocytic disorders.
Measure: Nature and Severity of Adverse Events Time: 12 monthsDescription: Response assessment using modified RECIST criteria for target lesions with specific mutations (e.g. BRAF-V600E) . Best response rate by 1 year of therapy.
Measure: Response assessment (Modified RECIST) of histiocytic lesions with specific mutations Time: 12 monthsAllocation: Non-Randomized
Parallel Assignment
There is one SNP
BRAF-V600E) . --- V600E ---
The purpose of this research study is to learn whether cobimetinib is safe and effective in subjects diagnosed with LCH, LCH-ND, RDD, JXG and ECD which may have a specfic mutation called BRAF-V600E. --- V600E ---
BRAF-V600E is a specific change in a gene that may cause cancer cells to grow and spread by sending constant signals to the MEK protein. --- V600E ---