Actively Recruiting
Binimetinib for People With Relapsed/Refractory BRAF Wild Type Hairy Cell Leukemia and Variant
Led by National Cancer Institute (NCI) · Updated on 2026-03-31
40
Participants Needed
1
Research Sites
394 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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....
CONDITIONS
Official Title
Binimetinib for People With Relapsed/Refractory BRAF Wild Type Hairy Cell Leukemia and Variant
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Histologically confirmed diagnosis of hairy cell leukemia (HCL) or HCL variant (HCLv) based on WHO criteria
- At least one indication for therapy such as low neutrophil count, low hemoglobin, low platelets, symptomatic enlarged spleen, enlarging HCL mass over 2 cm, high leukemia cell count, or fast leukemic doubling time
- Disease that is refractory (no response or progression within 1 year of first treatment) or relapsed after at least one prior purine analog treatment
- Confirmed BRAF wild type status from fresh bone marrow, blood, or lymph node samples
- Ineligible for or unable to access moxetumomab pasudotox trial at NCI
- Age 18 years or older
- ECOG performance status 0 to 2 (Karnofsky score 60% or higher)
- Adequate organ and marrow function as defined by specific liver, kidney, and blood test criteria
- Use of effective contraception for females of childbearing potential and non-sterilized males during and after treatment
- Willingness to discontinue breastfeeding during treatment and shortly after
- Ability to understand and sign informed consent
- Willingness to co-enroll in a related study for sample collection
You will not qualify if you...
- Chemotherapy, immunotherapy, or radiotherapy within 2 weeks before starting study treatment
- Prior treatment with binimetinib
- Use of other investigational agents within 14 days before starting treatment
- Major surgery within 6 weeks before treatment or not recovered from surgery side effects
- Known allergy or contraindication to binimetinib or its ingredients
- Inability to swallow or keep study drug down
- Pregnancy confirmed by positive pregnancy test
- Uncontrolled infections, cardiac dysfunction, pulmonary infections or edema, or psychiatric/social issues limiting study compliance
- Active hepatitis B or C infection (cleared infections allowed under certain conditions)
- Active second cancer requiring treatment (except minor skin cancers)
- HIV positive without appropriate treatment and CD4 count above 200
- History of bone marrow or stem cell transplant
- Significant cardiovascular disease or heart function problems
- Gastrointestinal issues affecting drug absorption
- Neuromuscular disorders with high creatinine kinase
- History or risk factors for retinal vein occlusion or certain eye diseases
- Recent thromboembolic or cerebrovascular events within 12 weeks (some stable cases allowed)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
H
Holly M Eager, R.N.
CONTACT
R
Robert J Kreitman, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here