Leukemia Clinical Trials

1,209 recruiting

Understanding Leukemia Clinical Trials

Imatinib (Gleevec), a drug that emerged from clinical trials in the early 2000s, transformed chronic myeloid leukemia from a near-fatal diagnosis into a manageable chronic condition, with survival rates exceeding 90% at five years. More recently, CAR-T cell therapies like tisagenlecleucel (Kymriah) — approved through landmark trials — have produced complete remissions in patients with aggressive forms of acute lymphoblastic leukemia who had exhausted all other options. Clinical trials remain the primary engine driving these breakthroughs in leukemia treatment, and many of tomorrow's standard therapies are available today only through trial participation.

Why Consider a Clinical Trial?

Leukemia encompasses several distinct diseases — acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myeloid leukemia (CML), and chronic lymphocytic leukemia (CLL) — each with different treatment landscapes and unmet needs. For some subtypes, particularly relapsed or refractory AML, standard chemotherapy regimens have not changed significantly in decades, and clinical trials may offer access to targeted therapies, bispecific antibodies, or novel combination regimens that are not yet available through routine care. Beyond access to new treatments, leukemia trials often include more frequent monitoring, specialized lab work, and closer follow-up than standard treatment plans. Many trials also cover the cost of the investigational drug and related procedures. For patients whose leukemia has returned after initial treatment or has not responded to frontline therapy, a clinical trial may represent the most promising path forward, giving access to drugs that specifically target the genetic mutations driving their particular form of the disease.

Frequently Asked Questions

Common questions about Leukemia clinical trials

Yes. Many leukemia trials are specifically designed for patients who have already undergone one or more lines of therapy. Some trials require that you have tried and not responded to certain treatments first. Your treatment history is one of the key factors used to determine which trials you may be eligible for.

Not always. Some trials use a randomized design where participants are assigned to either the new treatment or the current standard of care. However, leukemia trials rarely use placebos alone — you will almost always receive active treatment. The study team will explain the design before you consent.

The active treatment phase varies widely depending on the type of leukemia and the trial design, ranging from a few months to two or more years. After active treatment ends, most trials include a follow-up period where your health is monitored, which can last several additional years. The consent form will outline the expected time commitment.

Yes. Because leukemia is more common in older adults, many trials are specifically designed for patients over 60 or 65 who may not tolerate intensive chemotherapy. These trials often test lower-intensity regimens or targeted therapies with more manageable side effect profiles.

If your disease progresses during a trial, the study team will discuss your options, which may include switching to a different treatment arm within the trial, transitioning to another trial, or returning to standard care. Your safety is monitored continuously, and you can leave a trial at any time for any reason.

Showing 120 of 1,209 trials

Recruiting
Phase 2

Comparing Cytarabine + Daunorubicin Therapy Versus Cytarabine + Daunorubicin + Venetoclax Versus Venetoclax + Azacitidine in Younger Patients With Intermediate Risk AML (A MyeloMATCH Treatment Trial)

Acute Myeloid Leukemia
National Cancer Institute (NCI)153 enrolled175 locationsNCT05554393
Recruiting
Phase 3

Testing Early Treatment for Patients With High-Risk Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Leukemia (SLL), EVOLVE CLL/SLL Study

Chronic Lymphocytic LeukemiaSmall Lymphocytic Lymphoma
National Cancer Institute (NCI)247 enrolled627 locationsNCT04269902
Recruiting
Phase 2

Venetoclax and HMA Treatment of Older and Unfit Adults With FLT3 Mutated Acute Myeloid Leukemia (AML) (A MyeloMATCH Treatment Trial)

Acute Myeloid Leukemia
National Cancer Institute (NCI)147 enrolled218 locationsNCT06317649
Recruiting
Phase 2

Testing the Effects of Novel Therapeutics for Newly Diagnosed, Untreated Patients With High-Risk Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)

Acute Myeloid LeukemiaAcute Myeloid Leukemia Post Cytotoxic TherapyAcute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm+1 more
National Cancer Institute (NCI)335 enrolled216 locationsNCT05554406
Recruiting
Phase 1

TAK-243 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndromes With Increased Blasts

Recurrent Myelodysplastic SyndromeRefractory Myelodysplastic SyndromeRecurrent Acute Myeloid Leukemia+4 more
National Cancer Institute (NCI)42 enrolled5 locationsNCT03816319
Recruiting
Not Applicable

A Study to Evaluate Next-Generation Sequencing (NGS) Testing and Monitoring of B-cell Recovery to Guide Management Following Chimeric Antigen Receptor T-cell (CART) Induced Remission in Children and Young Adults With B Lineage Acute Lymphoblastic Leu...

Acute Lymphoblastic LeukemiaB-ALL
National Cancer Institute (NCI)60 enrolled8 locationsNCT05621291
Recruiting
Phase 1

Testing the Addition of an Anti-cancer Drug, SNDX-5613, to the Standard Chemotherapy Treatment (Daunorubicin and Cytarabine) for Newly Diagnosed Patients With Acute Myeloid Leukemia That Has Changes in NPM1 or MLL/KMT2A Gene

Acute Myeloid Leukemia With KMT2A RearrangementAcute Myeloid Leukemia With NPM1 Mutation
National Cancer Institute (NCI)28 enrolled22 locationsNCT05886049
Recruiting
Phase 3

A Study of BGB-16673 Compared to Investigator's Choice in Participants With Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma Previously Exposed to Both Bruton Tyrosine Kinase (BTK) and B-cell Leukemia/Lymphoma 2 Protein (BCL2) Inhibitors

Chronic Lymphocytic LeukemiaCLL
BeOne Medicines250 enrolled112 locationsNCT06846671
Recruiting
Phase 2

Testing the Addition of the Anti-cancer Drug Venetoclax and/or the Anti-cancer Immunotherapy Blinatumomab to the Usual Chemotherapy Treatment for Infants With Newly Diagnosed KMT2A-rearranged or KMT2A-non-rearranged Leukemia

Acute Leukemia of Ambiguous LineageB Acute Lymphoblastic Leukemia
National Cancer Institute (NCI)153 enrolled101 locationsNCT06317662
Recruiting
Phase 3

A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML)

Leukemia, Myeloid, Acute
Janssen Research & Development, LLC600 enrolled238 locationsNCT06852222
Recruiting
Phase 2

Venetoclax for the Treatment of Patients With Relapsed Hairy Cell Leukemia

Recurrent Hairy Cell LeukemiaRecurrent Hairy Cell Leukemia Variant
National Cancer Institute (NCI)20 enrolled21 locationsNCT06311227
Recruiting
Phase 2

Comparing New Treatments for People With Newly Diagnosed Acute Myeloid Leukemia That Has an IDH2 Gene Change (A MyeloMATCH Treatment Trial)

Acute Myeloid Leukemia
National Cancer Institute (NCI)93 enrolled112 locationsNCT06672146
Recruiting
Phase 1

Testing the Combination of an Anti-Cancer Drug, Iadademstat, With Other Anti-Cancer Drugs (Venetoclax and Azacitidine) for Treating AML

Acute Myeloid Leukemia
National Cancer Institute (NCI)45 enrolled4 locationsNCT06514261
Recruiting
Phase 2

MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)

Acute Myeloid LeukemiaMyelodysplastic SyndromeAcute Myeloid Leukemia Post Cytotoxic Therapy+2 more
National Cancer Institute (NCI)2,000 enrolled331 locationsNCT05564390
Recruiting
Phase 1

Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Hematologic Malignancies

Leukemia, Lymphocytic, AcuteBlood CancerMyeloid Leukemia, Acute+8 more
National Cancer Institute (NCI)86 enrolled1 locationNCT06904066
Recruiting
Phase 3

Testing the Use of Steroids and Tyrosine Kinase Inhibitors With Blinatumomab or Chemotherapy for Newly Diagnosed BCR-ABL-Positive Acute Lymphoblastic Leukemia in Adults

B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1
National Cancer Institute (NCI)348 enrolled211 locationsNCT04530565
Recruiting
Phase 1Phase 2

Testing the Combination of Anti-cancer Drugs, Tovorafenib Plus Rituximab, in Patients With Hairy Cell Leukemia

Hairy Cell LeukemiaRecurrent Hairy Cell LeukemiaRefractory Hairy Cell Leukemia
National Cancer Institute (NCI)84 enrolled1 locationNCT06965114
Recruiting
Phase 2

Testing the Effectiveness of the Anti-cancer Drug, Mirdametinib, in Treating Relapsed, Refractory Chronic Lymphocytic Leukemia

Recurrent Chronic Lymphocytic LeukemiaRecurrent Small Lymphocytic LymphomaRefractory Chronic Lymphocytic Leukemia+1 more
National Cancer Institute (NCI)20 enrolled1 locationNCT07061951
Recruiting
Phase 1Phase 2

A Dose-Escalation and Expansion Study of BGB-16673 in Participants With B-Cell Malignancies

Diffuse Large B Cell LymphomaChronic Lymphocytic LeukemiaNon-Hodgkin Lymphoma+6 more
BeOne Medicines614 enrolled120 locationsNCT05006716
Recruiting
Phase 2

Study of Zanubrutinib, Obinutuzumab, and Venetoclax in Patients With Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Leukemia (SLL)

Chronic Lymphocytic Leukemia (CLL)Small Lymphocytic Leukemia (SLL)
Memorial Sloan Kettering Cancer Center230 enrolled8 locationsNCT03824483