Actively Recruiting

Phase 2
Age: 18Years +
All Genders
NCT04324112

Encorafenib Plus Binimetinib for People With BRAF V600 Mutated Relapsed/Refractory HCL

Led by National Cancer Institute (NCI) · Updated on 2026-03-31

45

Participants Needed

1

Research Sites

404 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Background: Hairy cell leukemia (HCL) does not usually respond to chemotherapy. Most people with HCL have a BRAF gene mutation. This can increase the growth of cancer cells. Vemurafenib has been tested to treat these people. However, researchers think a combination of drugs might work better. Objective: To test if treatment with a combination of encorafenib and binimetinib in BRAF mutant HCL is more effective than treatment with vemurafenib. Eligibility: People ages 18 and older with BRAF mutant HCL that did not respond to or came back after treatment Design: Participants will be screened with: Medical history Physical exam Bone marrow biopsy: A needle will be injected through the participant s skin and into a bone to remove liquid. Blood and urine tests Heart and lung function tests CT or MRI scan: Participants will lie in a machine that takes pictures of the body. They may have a contrast agent injected into a vein. Eye exam Participants will take the study drugs by mouth in 28-day cycles. They will take encorafenib daily. They will take binimetinib twice daily. They will keep a pill diary. Participants will take their temperature daily. Participants will have at least 1 visit before each cycle. Visits will include repeats of some screening tests. They will also include abdominal ultrasounds, exercise stress tests, and skin evaluations. 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. Then they will have annual follow-ups....

CONDITIONS

Official Title

Encorafenib Plus Binimetinib for People With BRAF V600 Mutated Relapsed/Refractory HCL

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Histologically confirmed diagnosis of hairy cell leukemia with BRAF V600 mutation
  • Indications for therapy such as low neutrophil count, low hemoglobin, low platelets, symptomatic enlarged spleen, enlarging leukemia mass, or high leukemia cell count
  • At least 18 years of age
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
  • Adequate organ and marrow function as defined by specific laboratory values
  • Females of childbearing potential must use highly effective contraception; males must use condoms and avoid sperm donation during and after treatment
  • Willingness to discontinue nursing during study treatment
  • Ability to understand and sign informed consent
  • Agreement to co-enroll in related human sample collection study
Not Eligible

You will not qualify if you...

  • Chemotherapy, immunotherapy, investigational agent, or radiotherapy within 4 weeks prior to study treatment
  • Prior therapy with encorafenib or binimetinib
  • Use of other investigational agents within 14 days prior to treatment
  • Major surgery within 6 weeks prior to treatment or not recovered from surgery side effects
  • Known allergies or inability to take study drugs
  • Pregnancy or positive pregnancy test
  • Uncontrolled infections, cardiac dysfunction, pulmonary infection, or psychiatric/social issues limiting compliance
  • Active hepatitis B or C infection unless controlled
  • Active second cancers requiring treatment except minor skin cancers
  • HIV infection unless on treatment with adequate immune status
  • History of bone marrow or stem cell transplant
  • Chronic pancreatitis
  • Significant cardiovascular disease or arrhythmias
  • Gastrointestinal conditions impairing drug absorption
  • Certain neuromuscular disorders
  • History or risk factors for retinal vein occlusion or related eye conditions
  • Recent thromboembolic or cerebrovascular events within 12 weeks
  • Use of strong CYP3A4 inhibitors or inducers

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 1 location

1

National Institutes of Health Clinical Center

Bethesda, Maryland, United States, 20892

Actively Recruiting

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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

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