Binimetinib for People With Relapsed/Refractory BRAF Wild Type Hairy Cell Leukemia and Var
Background: Most people with hairy cell leukemia have a BRAF gene mutation. They can be treated with BRAF inhibitors, drugs that target this mutation. For people who do not have this mutation, BRAF inhibitors are not a treatment option. We found that in hairy cell leukemia, when BRAF is not mutated, the MEK gene freque
| Condition(s) | Hairy Cell Leukemia |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| Summary | Background: Most people with hairy cell leukemia have a BRAF gene mutation. They can be treated with BRAF inhibitors, drugs that target this mutation. For people who do not have this mutation, BRAF inhibitors are not a treatment option. We found that in hairy cell leukemia, when BRAF is not mutated, the MEK gene frequently is. Binimetinib is a MEK inhibitor which targets MEK. It is important to determine if this drug can be a good treatment option in those who cannot benefit treatment with BRAF inhibitors. Objective: To see if binimetinib is an effective treatment for hairy cell leukemia that does not have a BRAF mutation. Eligibility: People ages 18 and older with hairy cell leukemia without a mutation in the BRAF gene and whose disease either did not respond to treatment or came back aft |
| Who can participate | * INCLUSION CRITERIA: * Histologically confirmed diagnosis of HCL or HCLv according to morphological and immunophenotypic criteria of World Health Organization (WHO) classification \[WHO, 2008 revised 2016\] of lymphoid neoplasm. Participants should have at least one of the following indications for therapy: * Absolute neutrophil count (ANC) \<1 x10\^3/mcL * Hemoglobin \<10g/dL * Platelets\<100 x10\^3/mcL * Symptomatic splenomegaly * Enlarging HCL mass or bone lesion \> 2cm in short axis * Leukemia cell count \>5x10\^3/mcL * Leukemic doubling time \<6 months Participants who have eligible blood counts within 4 weeks prior to initiation of study therapy will not be considered ineligible if subsequent blood counts prior to initiation of study therapy fluctuate and become ineligible up until |
| Ages | 18 Years |
| Sex | All |
| Lead sponsor | National Cancer Institute (NCI) |
| Locations | Bethesda, Maryland, United States |
| Start date | 2021-01-07 |
| NCT ID | NCT04322383 |
| Official listing | https://clinicaltrials.gov/study/NCT04322383 |