NCT06311227

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

PRIMARY OBJECTIVE: I. To determine the objective response rate (ORR) of venetoclax. SECONDARY OBJECTIVES: I. 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: I. 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.

Conditions

• Recurrent Hairy Cell Leukemia
• Recurrent Hairy Cell Leukemia Variant

Interventions

Type	Name	Description
PROCEDURE	Biospecimen Collection	Undergo blood sample collection
PROCEDURE	Bone Marrow Aspiration	Undergo bone marrow aspiration
PROCEDURE	Bone Marrow Biopsy	Undergo bone marrow biopsy
PROCEDURE	Computed Tomography	Undergo CT scan
PROCEDURE	Lumbar Puncture	Undergo lumbar puncture
PROCEDURE	Magnetic Resonance Imaging	Undergo MRI
DRUG	Venetoclax	Given PO

Timeline

• Start date: 2024-12-16
• Primary completion: 2027-05-20
• Completion: 2027-05-20
• First posted: 2024-03-15
• Last updated: 2026-04-14

Locations

21 sites across 1 country: United States

Regulatory

• FDA-regulated drug study