A Phase 2 interventional study of CAV in Acute Myeloid Leukemia (AML), sponsored by University of Rochester. Not yet recruiting. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-02-20.
Sponsored by University of Rochester · Phase 2, Interventional, and Treatment
Acute myeloid leukemia (AML) is a bone marrow cancer that is challenging to treat. It is the most common type of acute leukemia, particularly in adults. There are around 20,000 cases of acute myeloid leukemia diagnosed in the United States every year. Despite the recent significant progress in the understanding of acute myeloid leukemia leading to the development of new therapies, significant challenges remain. The initial treatment for acute myeloid leukemia involves using therapies aimed at reducing the disease burden in the bone marrow to the lowest possible level (a state known as disease remission). This is usually followed by consolidation treatment aimed at curing the disease. The initial treatment involves high intensity chemotherapy in younger adults who can tolerate these therapies and low intensity therapies for older adults or those with other medical conditions that prohibit them from receiving high intensity chemotherapy. The consolidation therapy involves either more chemotherapy or a bone marrow transplant. In the recent years, a treatment regimen consisting of two drugs; Azacytidine and Venetoclax has become the standard of care for low intensity therapy intended for older adults. Despite significant improvement in outcomes of acute myeloid leukemia in older adults after the introduction of Azacytidine/Venetoclax, yet 40% of patients who receive this treatment will either be refractory to it or relapse after an initial remission. Those whose leukemia relapses after Azacytidine/Venetoclax treatment are left with very few treatment options and have a dismal prognosis. Based on previous laboratory studies, certain subtypes of acute myeloid leukemia tend to not respond as well to Azacytidine/Venetoclax therapy and have a better chance of responding to the treatment regimen the investigators are proposing in this study. The study treatment regimen consists of 3 drugs; Cladribine, low dose Cytarabine and Venetoclax. Demonstrating efficacy of the study regimen in treatment of relapsed/refractory acute myeloid leukemia, after prior Venetoclax therapy, will provide another treatment option for those with a relapsed/refractory disease who wish to continue receiving therapy.
Among the main mechanisms implicated in acute myeloid leukemia (AML) relapse after treatment with Venetoclax is upregulation of myeloid cell leukemia 1 (MCL-1) in AML cells with their survival becoming less dependent on the Venetoclax target, B-cell leukemia/lymphoma 2 (BCL-2). Leukemic stem cells (LSCs) in AML with monocytic differentiation are significantly more resistant to treatment with azacytidine/venetoclax being less dependent on BCL-2 and demonstrating preferential reliance on MCL-1 for survival. Cladribine, combined with Venetoclax in preclinical studies have shown efficacy in overcoming resistance to Venetoclax by downregulating MCL-1, particularly in AML with monocytic features and/or rat sarcoma oncogene (RAS) mutations.
The investigators propose to use the cladribine, low-dose cytarabine and venetoclax (CAV) regimen in patients with relapsed/refractory or secondary AML (transformed from myelodysplastic syndrome [MDS], myeloproliferative neoplasm [MPN] or MDS/MPN overlap) who were previously treated with a venetoclax-containing regimen, whose disease lacks molecular targets with approved therapies. CAV is a medium intensity regimen that has demonstrated acceptable safety and tolerability in older patients with AML and/or those who are unfit for intensive therapies
Objective:
The primary objective is to estimate the rate of composite complete remission (CRc) in participants with relapsed/refractory or secondary AML who are treated with cladribine, low-dose cytarabine and venetoclax after prior treatment with a venetoclax-containing regimen.
2,971 studies on the registry are indexed under Leukemia, Myeloid, Acute; 744 are open to participants now.
This study's planned enrollment of 24 is below the median of 41 across 2,508 interventional studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute studies →University of Rochester is the lead sponsor of 715 studies on the registry; 116 are open to participants now.
Of its 56 completed or terminated interventional studies of FDA-regulated products, 44 (79%) have results posted.
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Participants with relapsed/refractory AML (according to European LeukemiaNet 2022 Criteria) after treatment with a Venetoclax-containing regimen.
Participants must agree to take the following reproductive precautions:
Adequate organ function as follows:
Pregnant or breastfeeding women
cladribine, low-dose cytarabine, venetoclax.
Drug: CAV
cladribine 5 mg/m2 intravenous infusion daily on days 1-5, cytarabine 20 mg/m2 subcutaneous injection once daily on days 1-10, venetoclax 400 mg oral daily days 1-21.
Composite complete remission
Best response of complete remission, complete remission with incomplete hematologic recovery or morphologic leukemia-free state after 2 cycles of treatment.
Time frame: Response assessment will be done once at the end of cycle 1 and once at the end of cycle 2 (each cycle is 28 days).
Patient reported outcomes
Evaluation of impact of the study regimen on the quality of life of the study participants by asking the study participants to fill in the European Organization for Research and Treatment of Cancer Quality of Life Core Questionnaire (EORTC QLQ-C30). All of the questionnaire scales range in score from 0 (Worst) to 100 (Best).
Time frame: Once before study treatments and once at the end of cycle 2 of treatment or earlier if the participant goes off the study before the end of cycle 2 (each cycle is 28 days)
No study locations are listed for this record.
Plan to share: No
This study is not yet recruiting, as verified in Feb 2026. You cannot join it, but the record below documents what was studied.
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