Venetoclax for the Treatment of Patients With Relapsed Hairy Cell Leukemia
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorNational Cancer Institute (NCI)
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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)
Disqualifiers
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
Trial design
Design model
Single group
Treatments tested in this trial
Biospecimen Collection
Procedure/SurgeryUndergo blood sample collection
Bone Marrow Aspiration
Procedure/SurgeryUndergo bone marrow aspiration
Bone Marrow Biopsy
Procedure/SurgeryUndergo bone marrow biopsy
Computed Tomography
Procedure/SurgeryUndergo CT scan
Lumbar Puncture
Procedure/SurgeryUndergo lumbar puncture
Magnetic Resonance Imaging
Procedure/SurgeryUndergo MRI
Venetoclax
DrugGiven PO
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: Treatment (venetoclax)Experimental treatment 7 interventions
Trial outcomes
Primary outcomes
1
Objective response rate
Will be determined with 95% confidence intervals for each group.
Time frame
Up to 30 days after the last doseSecondary outcomes
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.
Time frame
Up to 30 days after the last dose2
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.
Time frame
Up to 30 days after the last dose3
MRD negativity
Will determine the rates of MRD-negative by blood flow cytometry.
Time frame
Up to 30 days after the last dose4
Incidence of adverse events
Time frame
Up to 30 days after the last doseOther outcomes
1
TP53 mutations and other mutations
Will correlate response to TP53 mutations and other mutations.
Time frame
Up to 30 days after the last dose2
Whole exome sequencing of relapsed HCL/HCLv samples
Will perform whole exome sequencing of relapsed HCL/HCLv samples to look for mutations, to correlate with response.
Time frame
Up to 30 days after the last doseSponsors and contacts
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Source ClinicalTrials.gov