Venetoclax for Relapsed Hairy Cell Leukemia
This study is testing a medication called venetoclax for people with hairy cell leukemia (HCL) or hairy cell leukemia variant (HCLv) that has returned after previous treatment. Venetoclax works by blocking a protein called Bcl-2, which helps cancer cells survive. The goal is to see how well venetoclax shrinks the cancer. You might be able to join if you are at least 18 years old, have HCL/HCLv that has come back, and haven't received venetoclax before. The study aims to enroll 20 participants and is currently unclear if it's recruiting.
- Study design
- This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 20 participants.
- What's involved
- You would take venetoclax by mouth daily for 28-day cycles, potentially for up to 19 cycles. You would also have CT scans, blood tests, and possibly bone marrow biopsies, aspirations, or lumbar punctures.
- Compensation
- Not stated in the trial record.
- Follow-up
- The main goal of the study is measured up to 30 days after your last dose of venetoclax.
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NCT06311227
Venetoclax for the Treatment of Patients With Relapsed Hairy Cell Leukemia
Recruiting
PHASE2Ages 18+InterventionalTreatmentNational Cancer Institute (NCI)Government-funded
~20 participants
Updated 2026-08-13 on ClinicalTrials.gov
What's tested:Biospecimen CollectionBone Marrow AspirationBone Marrow BiopsyComputed TomographyLumbar PunctureMagnetic Resonance Imaging
At a glance
Recruiting sites
19 of 21 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Objective response rate
Measured over Up to 30 days after the last dose
Conditions
Where it's being run
21 sites across 6 statesFlorida8
Kansas3
Missouri3
Ohio3
California2
Maryland2
Study leadership
- Robert J Kreitman · PRINCIPAL_INVESTIGATOR · National Cancer Institute LAO
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Patients must have histologically or cytologically confirmed HCL/HCLv after purine analog therapy who are relapsed from or are ineligible for BRAF therapy and have not received prior venetoclax
Age ≥ 18 years
Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 (Karnofsky ≥ 60%)
Total bilirubin ≤ 3 x institutional upper limit of normal (ULN) unless consistent with Gilbert's (ration between total and direct bilirubin \> 5)
Aspartate aminotransferase (AST)(serum glutamic oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase \[SGPT\]) ≤ 3 × institutional ULN
Serum creatinine ≤ 2.0 mg/dL OR creatinine clearance ≥ 45 mL/min/1.73m\^2
Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, patients should be class 2B or better
Patients must have had no HCL/HCLv treatment for ≥ 4 weeks prior to enrollment, and those with treatment \> 4 weeks prior to enrollment must not be responding to their last treatment with decreasing tumor burden or improving disease related cytopenias
Patients must have a need for treatment due to absolute neutrophil count (ANC) \< 1/nL, hemoglobin (Hgb) \< 10g/dL, platelets (Plt) \< 100/nL, symptomatic splenomegaly, HCL mass with short axis \> 2cm outside or \> 0.5 cm inside the CNS, HCL/HCLv count \> 5/nL in blood or \> 25/mm\^3 in cerebrospinal fluid (CSF), HCL/HCLv doubling time \< 6 months and increasing lytic or blastic bone lesions
The effects of venetoclax on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) during treatment and for 30 days after the last dose of venetoclax. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception during treatment and for 30 days after the last dose of venetoclax
Ability to understand and the willingness to sign a written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants
Ability and willingness to swallow pills
Exclusion
Patients who have received prior venetoclax
Patients who are receiving any other investigational agents
History of allergic reactions attributed to compounds of similar chemical or biologic composition to venetoclax
Patients with uncontrolled intercurrent illness or any other significant condition(s) that would make participation in this protocol unreasonably hazardous
Pregnant women are excluded from this study because venetoclax is a B-cell lymphoma-2 (BCL-2) inhibitor agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with venetoclax, breastfeeding should be discontinued if the mother is treated with venetoclax
Malabsorption syndrome or other conditions that would interfere with intestinal absorption
Live attenuated vaccines should not be administered within 4 weeks prior to, during, or 30 days after study treatment and recovery has occurred
What this trial measures
- Objective response rateUp to 30 days after the last dose
Will be determined with 95% confidence intervals for each group.