Leukemia is cancer of the blood-forming cells in bone marrow. It includes acute forms that progress quickly and chronic forms that develop slowly. Treatment ranges from chemotherapy to stem cell transplant to targeted drugs that exploit specific mutations in leukemia cells.
What's actually going on in research
Trials are testing CAR-T cell therapy for relapsed disease, bispecific antibodies that guide immune cells to kill leukemia, targeted drugs for specific mutations like FLT3 and IDH, and gentler chemotherapy regimens for older adults. Researchers are also studying minimal residual disease testing to detect relapse earlier and predict which patients need more aggressive treatment.
CAR-T cell therapy
CAR-T takes a patient's immune cells, engineers them to recognize leukemia, and infuses them back. It's FDA-approved for some types of relapsed B-cell leukemia and is being tested in earlier treatment lines.
Bispecific antibodies
These drugs connect a leukemia cell to a patient's T cell, triggering the immune system to attack. Blinatumomab is approved for some forms of acute lymphoblastic leukemia, and newer versions are in trials.
Targeted mutation drugs
Drugs like midostaurin and gilteritinib block specific mutations found in acute myeloid leukemia. Trials are testing combinations of these drugs with chemotherapy to improve outcomes.
What to know before you search
Eligibility typically depends on leukemia subtype, prior treatments, genetic mutations, fitness for intensive chemotherapy, and whether transplant has been attempted.
What types of trials are currently open
- Induction therapy trials — Testing new chemotherapy combinations or targeted drugs given right after diagnosis to put leukemia into remission.
- Maintenance therapy trials — Testing drugs given after remission to prevent relapse, often gentler treatments taken for months or years.
- Relapsed disease trials — Testing CAR-T, bispecific antibodies, or new drug combinations for leukemia that has come back or never fully responded.
- Transplant trials — Studies of stem cell transplant timing, donor matching, and drugs to prevent graft-versus-host disease.
- Minimal residual disease trials — Testing whether treating leukemia detected only by sensitive lab tests can prevent full relapse.
Recently added Leukemia trials
A Phase 2 Study of MAX-40279 Combined With Venetoclax and Azacitidine in Unfit Newly Diagnosed Acute Myeloid Leukemia
This is a prospective, multicenter, phase Ⅱ study consisting of dose escalation and ex-pansion stages in unfit newly diagnosed AML.
DEC3-VEN vs. Venetoclax-Based vs. DA Regimens in Newly Diagnosed AML
Protocol Title: A Prospective, Randomized Controlled Study of Venetoclax Plus 3-Day Decitabine (DEC3-VEN) Versus Venetoclax Plus "2+6" DA Versus "3+7" DA Regimen in Adult Patients with Newly Diagnosed Acute Myeloid Leukemia (AML) Objective: This is a prospective, open-label, randomized, multicenter, phase II trial designed to compare the efficacy and safety of three induction regimens-Venetoclax plus 3-day Decitabine (DEC3-VEN), Venetoclax plus "2+6" DA, and standard "3+7" DA-in adult patients with newly diagnosed, intensifiable AML. Study Design: The study employs a prospective, randomized, controlled, multicenter design. A total of 291 eligible patients will be enrolled across approximately 10 centers in China and randomized in a 2:2:1 ratio to the three treatment arms. Key Eligibility Criteria: Inclusion: Aged 16-65 with newly diagnosed non-APL AML (excluding favorable CBF-AML), eligible for intensive chemotherapy, ECOG ≤2, and adequate organ function. Exclusion: Prior AML treatment, secondary AML, active severe infection, significant cardiac comorbidity, or known hypersensitivity to the study drugs. Interventions: Induction: Arm A (VEN+"2+6"DA): Venetoclax (D1-8), Daunorubicin (D2-3), Cytarabine (D2-7). Arm B (DEC3-VEN): Venetoclax (D1-14), Decitabine (D4-6). FLT3/ITD+ patients add Sorafenib or Gilteritinib (D8-14). Arm C ("3+7"DA): Daunorubicin (D1-3), Cytarabine (D1-7). Consolidation (2 cycles): Arms A/B: Venetoclax + Intermediate-Dose Cytarabine. Arm C: High-Dose Cytarabine. Consolidation (Subsequent cycles): All arms receive multiple cycles of DA or HA regimens. Maintenance (6-8 cycles): Arms A/B: Venetoclax + Azacitidine. Arm C: Azacitidine. Main Outcome Measures: Primary Endpoint: Composite Complete Remission Rate (CR + CRi) after induction. Secondary Endpoints: Overall Survival (OS), CR rate, MRD-negative rate, Relapse-Free Survival (RFS), Event-Free Survival (EFS), and safety. Keywords: Acute Myeloid Leukemia, Venetoclax, Decitabine, Consolidation Therapy, Maintenance Therapy, Randomized Controlled Trial.
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