RecruitingPhase 2NCT04322383

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

Phase 2 Trial for Binimetinib for Patients With Relapsed/Refractory BRAF Wild Type Hairy Cell Leukemia and Variant


Sponsor

National Cancer Institute (NCI)

Enrollment

40 participants

Start Date

Jan 7, 2021

Study Type

INTERVENTIONAL

Conditions

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 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....


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a drug called binimetinib for people with a rare blood cancer called hairy cell leukemia (HCL) or a related variant (HCLv) that has come back or stopped responding to standard treatment. Importantly, this trial is specifically for patients whose cancer does NOT have a mutation called BRAF — a different biological subtype. **You may be eligible if...** - You have been diagnosed with hairy cell leukemia or its variant - Your cancer is confirmed to be BRAF wild-type (no BRAF mutation) - Your cancer has relapsed or stopped responding after at least one prior standard chemotherapy treatment - You have low blood counts, an enlarged spleen, or other signs that your leukemia is active and needs treatment - You are 18 years or older - You are unable, unwilling, or ineligible for another specific trial at the NCI **You may NOT be eligible if...** - Your cancer has the BRAF V600 mutation (a different trial may suit you better) - You have not yet tried standard chemotherapy - Your leukemia is well-controlled without clear signs of active disease Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGbinimetinib

Binimetinib will be given orally at a dose of 45mg BID continuously for 28-day cycles with no resting period between cycles.


Locations(1)

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

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NCT04322383


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