Phase 2 Trial of Oral Binimetinib for Adults With Relapsed or Refractory BRAF Wild Type Hairy Cell Leukemia and Variant

What this Trial Is About

Hairy cell leukemia HCL is a rare type of slow-growing B-cell leukemia. Most people with HCL have a BRAF gene mutation and can be treated with drugs targeting this mutation. However, some patients do not have this mutation and cannot use BRAF inhibitors. This trial investigates binimetinib, a drug that targets the MEK gene, to see if it can treat HCL patients without the BRAF mutation, especially those whose disease has returned or not responded to previous treatments. Participants will take binimetinib orally twice daily in 28-day cycles without breaks between cycles. The study is a single-group phase 2 trial aiming to measure how well binimetinib works in these patients. Before starting treatment, participants undergo various tests including medical history review, physical exams, blood and urine tests, heart and lung tests, eye exams, bone marrow biopsies, and imaging scans. Additional tests like abdominal ultrasound and exercise stress tests are done before treatment begins. Treatment continues as long as the disease does not worsen and side effects remain manageable. During the study, participants visit at least once before each treatment cycle for repeated tests to monitor their health and disease status. They keep a medication diary to track dosing. After the last dose, there is a follow-up visit about one month later, then yearly visits to assess overall response, survival, safety, and disease progression. Researchers also study how genetic mutations may affect treatment response. The study may last several years depending on individual participant outcomes.

Binimetinib for People With Relapsed/Refractory BRAF Wild Type Hairy Cell Leukemia and Variant