Skip to main content
An official website of the United States government
Government Funding Lapse
Because of a lapse in government funding, the information on this website may not be up to date, transactions submitted via the website may not be processed, and the agency may not be able to respond to inquiries until appropriations are enacted.

The NIH Clinical Center (the research hospital of NIH) is open. For more details about its operating status, please visit cc.nih.gov.

Updates regarding government operating status and resumption of normal operations can be found at opm.gov.

Avapritinib in Combination with Decitabine for the Treatment of Patients with Systemic Mastocytosis with an Associated Hematologic Neoplasm

Trial Status: active

This phase Ib trial tests the safety, best dose, and effectiveness of avapritinib in combination with decitabine or decitabine and cedazuridine in treating patients with systemic mastocytosis with an associated hematologic neoplasm (SM-AHN). Systemic mastocytosis is a rare disease in which too many mast cells (a type of immune system cell) are found in the skin, bones, joints, lymph nodes, liver, spleen, and gastrointestinal tract. Mast cells give off chemicals such as histamine that can cause flushing (a hot, red face), itching, abdominal cramps, muscle pain, nausea, vomiting, diarrhea, low blood pressure, and shock. Patients with SM-AHN have a hematologic malignancy as well as systemic mastocytosis. SM-AHN is a difficult disease to treat. Using a combination approach to treat both aspects of the disease has the potential to provide enhanced disease control; however, overlapping toxicity is a concern. Avapritinib is an experimental drug used to treat all forms of systemic mastocytosis. Decitabine, also called dacogen, is a drug used to treat adults with myelodysplastic syndromes, including chronic myelomonocytic leukemia. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Decitabine and cedazuridine is a combination of two drugs. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Giving avapritinib in combination with decitabine may be safe, tolerable and effective in treating patients with SM-AHN.