Venetoclax for the Treatment of Patients With Relapsed Hairy Cell Leukemia
A Phase 2 Study of Venetoclax in Relapsed Classic or Variant Hairy Cell Leukemia
2 other identifiers
interventional
20
1 country
21
Brief 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. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Dec 2024
21 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 13, 2024
CompletedFirst Posted
Study publicly available on registry
March 15, 2024
CompletedStudy Start
First participant enrolled
December 16, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 20, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
May 20, 2027
April 14, 2026
January 1, 2026
2.4 years
March 13, 2024
April 11, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Objective response rate
Will be determined with 95% confidence intervals for each group.
Up to 30 days after the last dose
Secondary Outcomes (6)
Complete remission (CR) rate
Up to 30 days after the last dose
Minimal residual disease (MRD)-negative CR rate
Up to 30 days after the last dose
MRD negativity
Up to 30 days after the last dose
Incidence of adverse events
Up to 30 days after the last dose
Complete response duration
Up to 30 days after the last dose
- +1 more secondary outcomes
Other Outcomes (2)
TP53 mutations and other mutations
Up to 30 days after the last dose
Whole exome sequencing of relapsed HCL/HCLv samples
Up to 30 days after the last dose
Study Arms (1)
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.
Interventions
Undergo bone marrow biopsy
Undergo blood sample collection
Undergo CT scan
Undergo MRI
Given PO
Eligibility Criteria
You may qualify if:
- 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
- +1 more criteria
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (21)
City of Hope Comprehensive Cancer Center
Duarte, California, 91010, United States
City of Hope at Irvine Lennar
Irvine, California, 92618, 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
University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami, Florida, 33136, United States
UM Sylvester Comprehensive Cancer Center at Kendall
Miami, Florida, 33176, United States
University of Kansas Cancer Center
Kansas City, Kansas, 66160, 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
NCI - Center for Cancer Research
Bethesda, Maryland, 20892, United States
University of Kansas Cancer Center - Briarcliff
Kansas City, Missouri, 64116, United States
University of Kansas Cancer Center - North
Kansas City, Missouri, 64154, United States
University of Kansas Cancer Center - Lee's Summit
Lee's Summit, Missouri, 64064, United States
University of Cincinnati Cancer Center-UC Medical Center
Cincinnati, Ohio, 45219, United States
Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210, United States
University of Cincinnati Cancer Center-West Chester
West Chester, Ohio, 45069, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Robert J Kreitman
National Cancer Institute LAO
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- NIH
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 13, 2024
First Posted
March 15, 2024
Study Start
December 16, 2024
Primary Completion (Estimated)
May 20, 2027
Study Completion (Estimated)
May 20, 2027
Last Updated
April 14, 2026
Record last verified: 2026-01
Data Sharing
- IPD Sharing
- Will share
"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."