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

Recruiting now · Phase 2

Conditions studied: Hairy Cell Leukemia

In brief

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 after treatment Design: Participants will be screened with: * Medical history * Physical exam * Blood and urine tests * Lung and heart tests * Eye exam * Bone marrow biopsy: A needle will be injected through the participant s skin into the bone to remove a sample of marrow. * CT or MRI scan: Participants will lie in a machine that takes pictures of the body. They might receive a contrast agent by vein. Before they start treatment, participants will have an abdominal ultrasound, pulmonary function tests, and exercise stress tests. Participants will take binimetinib by mouth twice daily in 28-day cycles. They will keep a medication diary. Participants will have at least one visit before every cycle. Visits will include repeats of some screening tests. Participants may continue treatment as long as their disease does not get worse and they do not have bad side effects. About a month after their last dose of treatment, participants will have a follow-up visit. They will then have visits once a year....

Key facts

Study ID

NCT04322383

Run by

National Cancer Institute (NCI)

People needed

40

Starts

2021-01-07

Expected to finish

2028-07-31

Last updated by the study team

2026-07-24

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Participants who have had chemotherapy, immunotherapy or radiotherapy within 2 weeks prior to the start of study treatment.
• Prior therapy with binimetinib.
• Participants who are receiving any other investigational agents or have received an investigational agent within 14 days prior to the start of study treatment.
• Participants who have undergone major surgery <= 6 weeks prior to start of study treatment or who have not recovered from side effects of such procedure.
• Known hypersensitivity or contraindication to any component of binimetinib or its excipients.
• Inability to swallow and retain study drug.
• Pregnant women as evaluated by a positive serum or urine beta-human chorionic gonadotropin (beta-hCG) test.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, cardiac disfunction (details as below), uncontrolled pulmonary infection, pulmonary edema or psychiatric illness/social situations that would limit compliance with study requirements.
• Evidence of active Hepatitis B Virus (HBV) or Hepatitis C Virus (HCV) infection.
• Note: Participants with laboratory evidence of cleared HBV or HCV infection may be enrolled. If positive for Hepatitis B core antibody or surface antigen, the participant must be on Tenofovir or Entecavir and Hepatitis B deoxyribonucleic acid (DNA) viral load (VL) must be <2000 IU/mL
• Active second malignancy requiring treatment other than minor resection of indolent cancers like basal cell and squamous skin cancers.
• Human immunodeficiency virus (HIV)-positive participants unless taking appropriate anti- HIV medications with a CD4 count of > 200. Otherwise, there may be an increased risk of infections.
• History of an allogeneic bone marrow or stem cell transplant.
• Impaired cardiovascular function or clinically significant cardiovascular disease including, but not limited to, any of the following:
• History of acute coronary syndromes (including myocardial infarction, unstable angina, coronary artery bypass grafting, coronary angioplasty or stenting) < 3 months prior to initiation of study therapy;
• Congestive heart failure requiring treatment (New York Heart Association Grade >= 2);
• Left ventricular ejection fraction (LVEF) < 50% as determined by multigated acquisition scan (MUGA) or transthoracic echocardiogram (TTE);
• Uncontrolled hypertension defined as persistent systolic blood pressure >=160 mmHg or diastolic blood pressure >= 100 mmHg despite current therapy;
• History or presence of clinically significant cardiac arrhythmias (including resting bradycardia, uncontrolled atrial fibrillation or uncontrolled paroxysmal supraventricular tachycardia);
• Triplicate average baseline QTcF interval >= 480 ms.
• Impairment of gastrointestinal function or disease which may significantly alter the absorption of study drug (e.g., active ulcerative disease, uncontrolled vomiting or diarrhea, malabsorption syndrome, small bowel resection with decreased intestinal absorption), or recent (<= 3 months) history of a partial or complete bowel obstruction, or other conditions that will interfere significantly with the absorption of oral drugs.
• Concurrent neuromuscular disorder that is associated with elevated creatinine kinase (CK) (e.g., inflammatory myopathies, muscular dystrophy, amyotrophic lateral sclerosis, spinal muscular atrophy).
• History or current evidence of retinal vein occlusion (RVO) or current risk factors for RVO (e.g., uncontrolled glaucoma or ocular hypertension, history of hyperviscosity or hypercoagulability syndromes); history of maculopathy or retinopathy for which there is an increased risk of
• MEK induced exudation (e.g., Central Serous Retinopathy).
• History of thromboembolic or cerebrovascular events <= 12 weeks prior to the first dose of study treatment. Examples include transient ischemic attacks, cerebrovascular accidents, hemodynamically significant (i.e., massive or sub-massive) deep vein thrombosis or pulmonary emboli.

Where it is running

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (enrolling)

Full record on ClinicalTrials.gov

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