New hope for rare leukemia: targeted pill tackles relapse

NCT ID NCT06311227

Disease control Sponsor: National Cancer Institute (NCI) Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Sep 10, 2026 · Updated 10 times

Summary

This study tests a pill called venetoclax for people whose hairy cell leukemia has returned after prior treatment. The drug blocks a protein that cancer cells need to survive. About 20 adults will take venetoclax to see if it shrinks tumors and improves blood counts, with the goal of controlling the disease long-term.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 20 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2024

Expected to finish

May 2027

An estimate. End dates often move.

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

hairy cell leukemia hairy cell leukemia variant refractory hairy cell leukemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Recurrent hairy cell leukemia Recurrent hairy cell leukemia variant

Contacts and locations

Locations

City of Hope Comprehensive Cancer Center

Duarte, California, 91010, United States
City of Hope at Irvine Lennar

Irvine, California, 92618, United States
NCI - Center for Cancer Research

Bethesda, Maryland, 20892, United States
Ohio State University Comprehensive Cancer Center

Columbus, Ohio, 43210, United States
UM Sylvester Comprehensive Cancer Center at Aventura

Aventura, Florida, 33180, United States
UM Sylvester Comprehensive Cancer Center at Coral Gables

Coral Gables, Florida, 33146, United States
UM Sylvester Comprehensive Cancer Center at Coral Springs

Coral Springs, Florida, 33065, United States
UM Sylvester Comprehensive Cancer Center at Deerfield Beach

Deerfield Beach, Florida, 33442, United States
UM Sylvester Comprehensive Cancer Center at Doral

Doral, Florida, 33166, United States
UM Sylvester Comprehensive Cancer Center at Hollywood

Hollywood, Florida, 33021, United States
UM Sylvester Comprehensive Cancer Center at Kendall

Miami, Florida, 33176, United States
University of Cincinnati Cancer Center-UC Medical Center

Cincinnati, Ohio, 45219, United States
University of Cincinnati Cancer Center-West Chester

West Chester, Ohio, 45069, United States
University of Kansas Cancer Center

Kansas City, Kansas, 66160, United States
University of Kansas Cancer Center - Briarcliff

Kansas City, Missouri, 64116, United States
University of Kansas Cancer Center - Lee's Summit

Lee's Summit, Missouri, 64064, United States
University of Kansas Cancer Center - North

Kansas City, Missouri, 64154, United States
University of Kansas Cancer Center-Overland Park

Overland Park, Kansas, 66210, United States
University of Kansas Hospital-Westwood Cancer Center

Westwood, Kansas, 66205, United States
University of Maryland/Greenebaum Cancer Center

Baltimore, Maryland, 21201, United States
University of Miami Miller School of Medicine-Sylvester Cancer Center

Miami, Florida, 33136, United States

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