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Recurrent Hairy Cell LeukemiaPhase 2NCT06311227

Venetoclax for the Treatment of Patients With Relapsed Hairy Cell Leukemia

A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Recurrent Hairy Cell Leukemia and Recurrent Hairy Cell Leukemia Variant, run by National Cancer Institute (NCI) at 21 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This phase II trial tests how well venetoclax works in treating patients with hairy cell leukemia that has come back after a period of improvement (relapsed). Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
21 sitesUnited States
Study dates
16 Dec 2024to 20 May 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 16Show fewer
  • 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

Who cannot join

  • 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

Trial locations and contacts

21 sites in 1 country.

21 sites in 1 country

United States21 sites

  • UM Sylvester Comprehensive Cancer Center at Aventura

    Aventura, Florida, 33180

  • University of Maryland/Greenebaum Cancer Center

    Baltimore, Maryland, 21201

  • NCI - Center for Cancer Research

    Bethesda, Maryland, 20892

  • University of Cincinnati Cancer Center-UC Medical Center

    Cincinnati, Ohio, 45219

  • Ohio State University Comprehensive Cancer Center

    Columbus, Ohio, 43210

  • UM Sylvester Comprehensive Cancer Center at Coral Gables

    Coral Gables, Florida, 33146

  • UM Sylvester Comprehensive Cancer Center at Coral Springs

    Coral Springs, Florida, 33065

  • UM Sylvester Comprehensive Cancer Center at Deerfield Beach

    Deerfield Beach, Florida, 33442

  • UM Sylvester Comprehensive Cancer Center at Doral

    Doral, Florida, 33166

  • City of Hope Comprehensive Cancer Center

    Duarte, California, 91010

  • UM Sylvester Comprehensive Cancer Center at Hollywood

    Hollywood, Florida, 33021

  • City of Hope at Irvine Lennar

    Irvine, California, 92618

  • University of Kansas Cancer Center

    Kansas City, Kansas, 66160

  • University of Kansas Cancer Center - Briarcliff

    Kansas City, Missouri, 64116

  • University of Kansas Cancer Center - North

    Kansas City, Missouri, 64154

  • University of Kansas Cancer Center - Lee's Summit

    Lee's Summit, Missouri, 64064

  • University of Miami Miller School of Medicine-Sylvester Cancer Center

    Miami, Florida, 33136

  • UM Sylvester Comprehensive Cancer Center at Kendall

    Miami, Florida, 33176

  • University of Kansas Cancer Center-Overland Park

    Overland Park, Kansas, 66210

  • University of Cincinnati Cancer Center-West Chester

    West Chester, Ohio, 45069

  • University of Kansas Hospital-Westwood Cancer Center

    Westwood, Kansas, 66205

Other trials in United States

About this study

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

This phase II trial tests how well venetoclax works in treating patients with hairy cell leukemia that has come back after a period of improvement (relapsed). Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival.

Read the detailed description

PRIMARY OBJECTIVE:

  1. To determine the objective response rate (ORR) of venetoclax.

SECONDARY OBJECTIVES:

  1. To determine the complete remission (CR) and minimal residual disease (MRD)-negative CR rates of venetoclax in relapsed hairy cell leukemia/hairy cell leukemia variant (HCL/HCLv).

II. To determine the rates of MRD-negative by blood flow cytometry with venetoclax.

III. To determine the safety of venetoclax in relapsed HCL/HCLv. IV. To determine the response and CR duration and MRD-negative survival in relapsed HCL/HCLv receiving venetoclax.

EXPLORATORY OBJECTIVES:

  1. To correlate response to TP53 mutations and other mutations, particularly for BRAF wild-type (WT) relapsed HCL/HCLv.

II. To perform whole exome sequencing (WES) of relapsed HCL/HCLv samples to look for mutations, to correlate with response.

OUTLINE:

Patients receive venetoclax orally (PO) once daily (QD) on days 1-28 of each cycle. Treatment repeats every 28 days for up to 19 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo computed tomography (CT) or magnetic resonance imaging (MRI) and blood sample collection throughout the study. Patients may undergo bone marrow biopsy and/or aspiration on study. Additionally, patients with known or suspected central nervous system (CNS) disease undergo lumbar puncture throughout the study.

After completion of study treatment, patients are followed up at 30 days.

Being tested
Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Computed Tomography, Lumbar Puncture
Medical subject headings
Leukemia, Hairy Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection

    Undergo blood sample collection

  • Bone Marrow AspirationProcedure or surgery

    Undergo bone marrow aspiration

  • Bone Marrow BiopsyProcedure or surgery

    Also called Biopsy of Bone Marrow, Biopsy, Bone Marrow

    Undergo bone marrow biopsy

  • Computed TomographyProcedure or surgery

    Also called CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography

    Undergo CT scan

  • Lumbar PunctureProcedure or surgery

    Also called LP, Spinal Tap

    Undergo lumbar puncture

  • Magnetic Resonance ImagingProcedure or surgery

    Also called Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan

    Undergo MRI

  • VenetoclaxDrug

    Also called ABT 199, ABT-0199, ABT-199, ABT199

    Given PO

Study groups in NCT06311227
Study groupRoleWhat they receive
Treatment (venetoclax)ExperimentalPatients receive venetoclax PO QD on days 1-28 of each cycle. Treatment repeats every 28 days for up to 19 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo CT or MRI and blood sample collection throughout the study. Patients may undergo bone marrow biopsy and/or aspiration on study. Additionally, patients with known or suspected CNS disease undergo lumbar puncture throughout the study.
Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Procedure: Computed Tomography; Procedure: Lumbar Puncture; Procedure: Magnetic Resonance Imaging; Drug: Venetoclax

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Objective response rate

    Will be determined with 95% confidence intervals for each group.

    Measured: Up to 30 days after the last dose

Secondary outcomes (6)

  1. Complete remission (CR) rate

    CR will be defined as resolution of cytopenias with neutrophils > 1.5/nL, platelets > 100/nL, and hemoglobin > 11g/dL at least 4 weeks from last transfusion or growth factor, resolution of splenomegaly by exam or by spleen size =< 17 cm by imaging, resolution of hairy cell leukemia (HCL)/hairy cell leukemia variant (HCLv)-related lymphadenopathy/masses to =< 2cm in short axis (=< 0.5 cm in short axis in the central nervous system [CNS]), and absence of morphological evidence of HCL/HCLv in the blood and bone marrow.

    Measured: Up to 30 days after the last dose

  2. Minimal residual disease (MRD)-negative CR rate

    Defined as meeting the criteria for CR plus absence of HCL/HCLv cells in the bone marrow aspirate and blood by flow cytometry, and negative immunochemistry of the bone marrow biopsy. Positive IHC involves B-cells > T-cells and most of the B-cells being consistent with HCL. Patients with CNS disease who achieve CR with positive cerebrospinal fluid flow cytometry will be considered CR with MRD.

    Measured: Up to 30 days after the last dose

  3. MRD negativity

    Will determine the rates of MRD-negative by blood flow cytometry.

    Measured: Up to 30 days after the last dose

  4. Incidence of adverse events

    Measured: Up to 30 days after the last dose

  5. Complete response duration

    Measured: Up to 30 days after the last dose

  6. MRD-negative survival

    Measured: Up to 30 days after the last dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Mar 2024
  2. Study start16 Dec 2024
  3. Primary completion20 May 2027 (estimated)
  4. Study completion20 May 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
20 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06311227
Sponsor's protocol ID
NCI-2024-01904
Other IDs
NCI-2024-01904, 10637, 10637
Status last verified
Sep 2026
First submitted
13 Mar 2024
Last updated
9 Sep 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: "NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page."

Publications

  • Zent CS, Tiacci E, Kreitman RJ, Tadmor T, Tallman MS, Wormann B, Andritsos LA, Arons E, Banerji V, Barrientos JC, Bhat SA, Blachly JS, Broccoli A, Call TG, Dearden C, Demeter J, Dietrich S, El-Sharkawi D, Fagarasanu A, Falini B, Forconi F, Gerrie AS, Gladstone DE, Gozzetti A, Hampel PJ, Hermel DJ, Iyengar S, Johnston JB, Juliusson G, Kipps TJ, Lauria F, Lozanski G, Parikh SA, Park JH, Polliack A, Quest G, Rai K, Ravandi F, Robak T, Rogers KA, Saven A, Seymour JF, Tam CS, Troussard X, Zenz T, Zinzani PL, Grever MR. Updated consensus guidelines for the diagnosis and management of patients with HCL and HCL variant. Blood. 2026 Jul 2;148(1):31-42. doi: 10.1182/blood.2025032757.

    PubMed 41980020

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Recurrent Hairy Cell Leukemia and Recurrent Hairy Cell Leukemia Variant, run by National Cancer Institute (NCI) at 21 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 16 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 21 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Objective response rate (time frame: Up to 30 days after the last dose). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06311227, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Sep 2026.

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