Binimetinib for Treating Hairy Cell Leukemia Without BRAF Mutation

Sponsor: National Cancer Institute (NCI)

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT04322383

Provider View

This study is testing if the drug binimetinib can be an effective treatment for people with hairy cell leukemia that does not have a BRAF mutation and has either not responded to previous treatments or has returned after treatment.

Patient Parameters

Program Overview

Most people with hairy cell leukemia have a mutation in the BRAF gene, which can be treated with specific drugs. However, for those without this mutation, these drugs are not an option. Researchers have found that in these cases, the MEK gene is often mutated. Binimetinib is a drug that targets the MEK gene, and this study aims to see if it can be a good treatment for those who cannot use BRAF inhibitors.

Description

Hairy cell leukemia (HCL) is a rare type of blood cancer. Most cases have a mutation in the BRAF gene, but some do not. These cases, known as BRAF wild type (WT) HCL, are more aggressive and harder to treat. Binimetinib is a drug that targets another gene, MEK, which is often mutated in BRAF WT HCL. This study is testing if binimetinib can help treat BRAF WT HCL.

Participants will take binimetinib by mouth twice daily in 28-day cycles. They will keep a medication diary and have regular visits to monitor their health and the treatment's effects. The study will continue as long as the disease does not worsen and side effects are manageable. After stopping treatment, participants will have follow-up visits to monitor their health.

The study is open to adults aged 18 and older with BRAF WT HCL that has not responded to previous treatments or has returned after treatment. Participants must have certain blood counts and organ functions and must not have uncontrolled infections or other serious health issues. Women of childbearing potential must use effective contraception during the study and for 30 days after the last dose of binimetinib.

Eligibility Criteria

Inclusion Criteria

  • Diagnosed with hairy cell leukemia (HCL) or its variant (HCLv) without a BRAF mutation.
  • Have received at least one prior treatment with purine analogs.
  • Need treatment due to low blood counts, enlarged spleen, or growing cancer mass.
  • Aged 18 years or older.
  • Good overall health with adequate organ function.
  • Willing to use effective contraception if of childbearing potential.
  • Able to understand and sign a consent form.
  • Must also enroll in a related study for sample collection.

Exclusion Criteria

  • Recent chemotherapy, immunotherapy, or radiotherapy within 4 weeks before starting the study.
  • Previous treatment with binimetinib.
  • Participation in another clinical trial within 14 days before starting the study.
  • Recent major surgery or not fully recovered from surgery.
  • Allergies to binimetinib or its ingredients.
  • Unable to swallow the study drug.
  • Pregnant or breastfeeding.
  • Uncontrolled health issues, such as infections or heart problems.
  • Active hepatitis B or C infection.
  • Other active cancers requiring treatment.
  • HIV-positive unless on treatment with a CD4 count over 200.
  • History of bone marrow or stem cell transplant.
  • History of pancreatitis or significant heart disease.
  • Gastrointestinal issues affecting drug absorption.
  • Neuromuscular disorders with elevated CK levels.
  • Eye conditions at risk from MEK inhibitors.
  • Recent blood clots or strokes unless stable on anticoagulants.

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