A Phase II Study of Cobimetinib in Patients with Histiocytic Disorders


Full Title

Phase II Trial of Single-agent Cobimetinib for Adults with Histiocytic Disorders


Histiocytosis is a general name for a group of diseases that involve an abnormal increase in the number of immune cells called histiocytes. Examples include Erdheim-Chester disease, Langerhans cell histiocytosis, Rosai-Dorfman disease, and histiocytic sarcoma. These diseases are treated with a variety of medications (including chemotherapy and medications that alter the immune system). Some forms of these diseases are mild, while others can be severe and difficult to treat.

In this study, researchers are evaluating the safety and effectiveness of treatment with the drug cobimetinib in patients with histiocytic disorders. Cobimetinib works by inhibiting MEK, a protein which can cause histiocytic tumors to grow. It is approved for treating advanced melanoma; its use in this study is considered investigational. Cobimetinib is taken orally (by mouth).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have a histiocytic disorder. In most cases, patients can participate whether or not they’ve already received treatment for their histiocytic disorder.
  • Patients may not have had prior treatments that target MEK.
  • Patients must be able to attend doctors’ visits at Memorial Sloan Kettering once per month.
  • This study is for patients age 16 and older.

For more information about this study, please contact Dr. Eli Diamond at 212-610-0243.





Disease Status

Newly Diagnosed