The following clinical trials represent a selection of key studies currently recruiting patients with leukemia for inclusion in investigations of new therapies and new regimens of existing treatments for the disease. Each clinical trial description includes the NLM Identifier to be used as a reference with ClinicalTrials.gov. This information can help oncology practice managers and providers direct eligible patients to one of these clinical trials.
1 Acalabrutinib for Untreated and Relapsed or Refractory CLL
The purpose of this open-label, multicenter, single-arm phase 3b study is to evaluate the safety and efficacy of acalabrutinib (Calquence) in the treatment of patients with treatment-naïve chronic lymphocytic leukemia (CLL), relapsed or refractory CLL, and patients previously treated with Bruton tyrosine kinase inhibitor. Patients aged ≥18 years with active CLL per ≥1 International Workshop on CLL (iwCLL) 2018 criteria, who have an Eastern Cooperative Oncology Group (ECOG) performance status ≤2, who have had fluorescence in situ hybridization within 60 days before or during screening, and who have had molecular analysis to detect IGHV mutation status at any time since diagnosis may be eligible if other criteria are met. Eligible patients will receive acalabrutinib 100 mg orally (PO) twice daily.
The primary outcome measure is the number of participants with adverse events (AEs) from screening to safety follow-up period (approximately 30 days from last dose). Secondary outcome measures include objective response rate (ORR), duration of response (DOR), and progression-free survival (PFS). Other outcome measures include overall survival (OS), time to next treatment, Patient-Reported Outcomes of the Common Terminology Criteria for Adverse Events up to 48 cycles, and patient-reported symptoms and health-related quality of life per European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC-QLQ-C30). The study plans to enroll 560 participants throughout the United States and worldwide. For more information, contact the AstraZeneca Clinical Study Information Center at 1-877-240-9479 or
2 Standard Chemotherapy versus Daunorubicin-Cytarabine with or without Gilteritinib in Newly Diagnosed AML
The purpose of this randomized phase 3 study is to compare the efficacy and safety of standard chemotherapy versus treatment with daunorubicin-cytarabine (Vyxeos) with or without gilteritinib (Xospata) in patients with newly diagnosed acute myeloid leukemia (AML) with or without FLT3 mutations. Patients aged ≤22 years with de novo AML with or without extramedullary disease according to the 2016 World Health Organization classification, who do not have any congenital long QT syndrome or heart block, and who do not have a known history of neurodevelopmental disorder prior to AML diagnosis may be eligible if other criteria are met. Eligible patients will be randomized to receive standard chemotherapy, daunorubicin-cytarabine plus gilteritinib, or daunorubicin-cytarabine alone.
The primary outcome is 3-year event-free survival, defined as the time from study entry until induction failure, relapse, secondary malignancy, or death. Secondary outcome measures include 3-year OS, proportion of patients positive for minimal residual disease (MRD), relapse rate, treatment-related mortality rate, and incidence of AEs. Other outcome measures include course duration, length of hospitalization, and time to count recovery. The study plans to enroll 1400 participants throughout the United States, Canada, and Puerto Rico. For more information, contact each site directly. The NLM identifier is NCT04293562.
3 Blinatumomab plus Chemotherapy in Newly Diagnosed B-Lymphoblastic Leukemia
The purpose of this randomized phase 3 study is to evaluate the efficacy of adding blinatumomab (Blincyto) to chemotherapy for the treatment of patients with newly diagnosed, standard-risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down syndrome. Patients aged 1 to 31 years with >25% blasts on bone marrow aspirate, who have no secondary hematologic malignancy, who do not require radiation therapy at diagnosis, and who have not received any previous cytotoxic chemotherapy treatment may be eligible if other criteria are met. Eligible patients will be randomized to receive standard chemotherapy with blinatumomab or chemotherapy alone.
The primary outcome measures are disease-free survival (DFS) in randomization-eligible patients with higher-risk features or standard-risk average features and DFS in boys with or without Down syndrome. Secondary outcome measures include DFS in patients with MRD-negative disease, DFS in patients with localized B-lymphoblastic lymphoma receiving standard therapy, change in neurocognitive functioning from baseline to end of therapy, and caregiver burden. Other outcome measures include prevalence of minimal marrow disease and neurocognitive, functional, and quality-of-life outcomes in patients with Down syndrome and B-lymphoblastic leukemia. The study plans to enroll 6720 participants throughout the United States and worldwide. For more information, contact the recruiting sites directly. The NLM identifier is NCT03914625.
4 Zanubrutinib versus Bendamustine plus Rituximab Combination in Previously Untreated CLL or SLL
The purpose of this global, open-label, randomized phase 3 study is to evaluate the efficacy of zanubrutinib (Brukinsa) versus the combination of bendamustine (Bendeka) plus rituximab (Rituxan) for the treatment of patients with previously untreated CLL or small lymphocytic lymphoma (SLL) regardless of 17p deletion status. Patients aged ≥18 years with a confirmed diagnosis of CD20-positive CLL or SLL; who have an ECOG performance status of 0-2; whose disease is unsuitable for chemoimmunotherapy; and who have adequate bone marrow, renal, and hepatic function may be eligible if other criteria are met. Eligible patients will be placed into 1 of 3 cohorts. Cohort 1 will be randomized in a 1:1 ratio to 2 zanubrutinib 80-mg capsules PO twice daily until disease progression or unacceptable toxicity or bendamustine 90 mg/m2 intravenously (IV) daily for the first 2 days of each cycle for 6 cycles plus rituximab 375 mg/m2 IV on day 0 of cycle 1 followed by 500 mg/m2 on day 1 of cycles 2 to 6. Cohort 2 will receive zanubrutinib monotherapy. Cohort 3 will receive zanubrutinib plus venetoclax 400 mg PO once daily.
The primary outcome measure is PFS between treatment groups in cohort 1, as determined by independent central review. The secondary outcome measures for each cohort include ORR, OS, and DOR between treatment groups (cohort 1); ORR, PFS, and DOR between treatment groups (cohort 2); and PFS, DOR, and rate of undetectable MRD between treatment groups (cohort 3). Other secondary outcomes include the number of participants having AEs, serious AEs, and the apparent rate of zanubrutinib clearance from plasma. The study plans to enroll 710 participants throughout the United States and worldwide. For more information, contact BeiGene at 1-877-828-5568 or
5 Venetoclax Added to Ibrutinib plus Obinutuzumab in Untreated, Older Patients with CLL
The purpose of this randomized phase 3 study is to compare the addition of venetoclax (Venclexta) to the standard treatment of ibrutinib (Imbruvica) plus obinutuzumab (Gazyva) in previously untreated older adult patients with CLL, and to investigate whether patients who receive the triplet therapy have no detectable disease after 1 year of treatment and can stop taking ibrutinib. Patients aged ≥70 years who have been diagnosed with intermediate- or high-risk CLL or SLL according to iwCLL 2018 criteria, who have an ECOG performance status of 0-2, who have adequate gastrointestinal absorption, and who are able to receive prophylactic treatment with either a xanthine oxidase inhibitor or rasburicase for tumor lysis syndrome may be eligible if other criteria are met. Eligible patients will be randomized to receive ibrutinib PO once daily plus obinutuzumab IV on days 1, 2, 8, and 15 of cycle 1, and on day 1 of cycles 2 to 6 for up to 14 cycles; or the same combination plus venetoclax PO once daily starting on cycle 3 for 14 cycles in the absence of disease progression or unacceptable toxicity.
The primary outcome measure is to compare the PFS between control treatment and experimental treatment strategies: ibrutinib plus obinutuzumab with ibrutinib maintenance versus ibrutinib/venetoclax/obinutuzumab regardless of ibrutinib maintenance or observation. Secondary outcome measures include bone marrow MRD complete response rate in each treatment arm, OS defined as the time from randomization date until death from any cause, and incidence of AEs. The study plans to enroll 454 participants throughout the United States and worldwide. For more information, contact Jennifer A. Woyach, MD, at the Alliance for Clinical Trials in Oncology at
6 BSC with or without Venetoclax plus Azacitidine as Maintenance in AML After First Remission
The purpose of this randomized, open-label, multicenter, phase 3 study is to evaluate the safety and efficacy of venetoclax (Venclexta) when added to azacitidine and best supportive care (BSC) versus BSC alone as maintenance therapy in adult patients with AML in first remission after conventional chemotherapy. Patients aged ≥18 years with newly diagnosed AML as confirmed by the World Health Organization 2016 criteria or who are in complete remission with incomplete blood recovery following completion of planned induction and consolidation chemotherapy, who have achieved first complete remission within 4 months of enrollment and are no more than 75 days since last dose of conventional therapy, who have an ECOG performance status ≤2, and whose disease has intermediate- or poor-risk cytogenetics per the National Comprehensive Cancer Network 2016 criteria may be eligible if other criteria are met. Patients will receive venetoclax and azacitidine or BSC for approximately 2 years with study visits varying from 1 to 5 per month. This study will be conducted in 3 parts. Part 1 will be the dose confirmation portion to determine the recommended phase 3 dose of venetoclax in combination with azacitidine. Part 2 will be the randomization portion to evaluate whether venetoclax in combination with azacitadine as maintenance therapy improves relapse-free survival compared with BSC. Part 3 will be the dose-finding portion to determine levels of venetoclax in combination with azacitidine to be explored.
The primary outcome measures include the number of participants with dose-limiting toxicities in part 1 of the study, relapse-free survival in part 2 of the study, and the number of participants with dose-limiting toxicities from the combination treatment in part 3 of the study. Secondary outcome measures include OS from date of randomization to date of death, percentage of patients who achieve minimal MRD, time to deterioration in global health status/quality of life based on EORTC-QLQ-C30, and change from baseline in patient-reported outcomes. This study plans to enroll 360 participants throughout the United States and worldwide. For more information, contact AbbVie Call Center at 1-844-663-3742, or
7 Steroids plus TKIs with Blinatumomab or Chemotherapy in Newly Diagnosed BCR-ABL–Positive B-Cell ALL
The purpose of this randomized phase 3 trial is to compare the efficacy of adding blinatumomab (Blincyto) to the standard treatment of chemotherapy plus steroids plus a tyrosine kinase inhibitor (TKI) versus standard treatment alone in patients with newly diagnosed BCR-ABL–positive B-cell acute lymphoblastic leukemia (ALL). Patients aged 18 to 75 years with centrally confirmed BCR-ABL translocation and B-cell ALL who have not received previous TKI treatment (or who have received <14 days of treatment), who have acute organ dysfunction at registration that is attributed to leukemia, and who have had a diagnosis of Philadelphia chromosome–positive ALL that has been confirmed locally may be eligible if other criteria are met. Eligible patients will be randomized to receive prednisone plus TKI treatment with ponatinib (Iclusig) or dasatinib (Sprycel) once daily on days 1 to 21; chemotherapy plus dexamethasone plus TKI treatment; or dexamethasone plus TKI treatment plus blinatumomab with or without chemotherapy.
The primary outcome measure is OS, defined as time between randomization and death from any cause, assessed up to 10 years from the date of registration. Secondary outcome measures include the rate of MRD negativity at week 15 and after re-induction, event-free survival from time at randomization to failure to achieve induction molecular remission by week 15, and incidence of AEs. The study plans to enroll 330 patients throughout the United States and worldwide. For more information, contact the recruiting sites directly. The NLM identifier is NCT04530565.