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Using venetoclax to treat relapsed hairy cell leukemia

A Phase 2 Study of Venetoclax in Relapsed Classic or Variant Hairy Cell Leukemia

Phase 2 Interventional National Cancer Institute (NCI) · NCT06311227

This study is testing if a medication called venetoclax can help people with relapsed hairy cell leukemia feel better and achieve remission.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment20 (estimated)
Ages18 Years and up
SexAll
SponsorNational Cancer Institute (NCI) NIH
Locations21 sites (Duarte, California and 20 other locations)
Trial IDNCT06311227 on ClinicalTrials.gov

What this trial studies

This phase II trial evaluates the effectiveness of venetoclax, a BCL-2 inhibitor, in patients with relapsed hairy cell leukemia (HCL) or its variant. The study aims to determine the objective response rate, complete remission rates, and safety of venetoclax in this patient population. Participants will receive oral venetoclax daily for up to 19 cycles, with regular monitoring through imaging and blood tests. Additionally, the study will explore genetic mutations that may correlate with treatment response.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with confirmed relapsed hairy cell leukemia who have not previously received venetoclax.

Not a fit: Patients who have not been treated with purine analog therapy or those who are not relapsed or ineligible for BRAF therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new effective option for patients with relapsed hairy cell leukemia.

How similar studies have performed: Other studies involving BCL-2 inhibitors have shown promising results in treating various hematological malignancies, suggesting potential success for this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* 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 Criteria:

* 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

Where this trial is running

Duarte, California and 20 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Recurrent Hairy Cell LeukemiaRecurrent Hairy Cell Leukemia Variant
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.