Leukemia Clinical Trials

97 recruiting

Leukemia Trials at a Glance

1,196 actively recruiting trials for leukemia are listed on ClinicalTrialsFinder across 6 cities in 70 countries. The largest study group is Phase 2 with 498 trials, with the heaviest enrollment activity in Houston, New York, and Boston. Lead sponsors running leukemia studies include M.D. Anderson Cancer Center, The First Affiliated Hospital of Soochow University, and Institute of Hematology & Blood Diseases Hospital, China.

Treatments under study

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 1,196 trials

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 enrolled346 locationsNCT05564390
Recruiting

Natural History Study of Monoclonal B Cell Lymphocytosis (MBL), Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Lymphoplasmacytic Lymphoma (LPL)/Waldenstrom Macroglobulinemia (WM), and Splenic Marginal Zone Lymphoma (SMZL)

Small Lymphocytic LymphomaWaldenstrom MacroglobulinemiaLymphoplasmacytic Lymphoma+2 more
National Heart, Lung, and Blood Institute (NHLBI)1,000 enrolled1 locationNCT00923507
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 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 enrolled629 locationsNCT04269902
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 enrolled177 locationsNCT05554393
Recruiting
Phase 1Phase 2

AZD0486 as Monotherapy in B-cell Acute Lymphoblastic Leukaemia

B-cell Acute Lymphoblastic Leukemia (B-ALL)
AstraZeneca163 enrolled80 locationsNCT06137118
Recruiting

Long-term Evaluation and Follow-up Care of Patients Treated With Stem Cell Transplants

Graft vs Host DiseaseGraft-versus-leukemiaGraft Rejection
National Heart, Lung, and Blood Institute (NHLBI)1,000 enrolled1 locationNCT00106925
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 enrolled220 locationsNCT05554406
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 enrolled220 locationsNCT06317649
Recruiting
Phase 2

Inotuzumab Ozogamicin and Blinatumomab With or Without Ponatinib in Treating Patients With Newly Diagnosed, Recurrent, or Refractory CD22-Positive B-Lineage Acute Lymphoblastic Leukemia

Recurrent B Acute Lymphoblastic LeukemiaB Acute Lymphoblastic Leukemia, Philadelphia Chromosome NegativeRefractory B Acute Lymphoblastic Leukemia
National Cancer Institute (NCI)84 enrolled274 locationsNCT03739814
Recruiting

Cognitive Aftereffects of Neurotoxicity in Children and Young Adults With Relapsed/Refractory Hematologic Malignancies Who Receive CAR T-cell Therapy

LymphomaLeukemia
National Cancer Institute (NCI)60 enrolled3 locationsNCT05237986
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 enrolled4 locationsNCT06965114
Recruiting
Phase 1

Study to Evaluate the Pharmacokinetics and Safety of Oral Decitabine and Cedazuridine in Cancer Patients With Renal Impairment

Acute Myeloid LeukemiaMyelodysplastic Syndromes
Taiho Oncology, Inc.18 enrolled21 locationsNCT04953897
Recruiting
Phase 1Phase 2

A Study of Subcutaneous Blinatumomab Administration in Participants With R/R and MRD+ B-ALL

B Cell Precursor Acute Lymphoblastic Leukemia
Amgen281 enrolled109 locationsNCT04521231
Recruiting
Phase 2Phase 3

A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)

Acute Myeloid LeukemiaMyelodysplastic SyndromesChronic Myelomonocytic Leukemia+1 more
Taiho Oncology, Inc.316 enrolled71 locationsNCT04256317
Recruiting
Phase 1

Study to Evaluate the Pharmacokinetics and Safety of Oral Decitabine and Cedazuridine in Cancer Patients With Hepatic Impairment

Acute Myeloid LeukemiaMyelodysplastic Syndromes
Taiho Oncology, Inc.27 enrolled22 locationsNCT04953910
Recruiting
Phase 2

Time-limited Triplet Combination of Pirtobrutinib, Venetoclax, and Obinutuzumab for Patients With Treatment-naïve Chronic Lymphocytic Leukemia (CLL) or Richter Transformation (RT)

Leukemia
M.D. Anderson Cancer Center60 enrolled1 locationNCT05536349
Recruiting
Phase 2

Adding Dasatinib Or Venetoclax To Improve Responses In Children With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (ALL) Or Lymphoma (T-LLY) Or Mixed Phenotype Acute Leukemia (MPAL)

T-cell LymphomaT-cell Acute Lymphoblastic LeukemiaMixed Phenotype Acute Leukemia
St. Jude Children's Research Hospital100 enrolled3 locationsNCT06390319
Recruiting

Study of Individuals and Families With Aberrations in DDX41 or Similar Cancer Predisposition Variants

Acute Myeloid LeukemiaMyelodysplastic SyndromesGermline Mutation
National Cancer Institute (NCI)510 enrolled1 locationNCT07019155
Recruiting
Phase 4

Identification of Necessary Information for Treatment Induction in Newly Diagnosed Acute Lymphoblastic Leukemia/Lymphoma

Acute Lymphoblastic LeukemiaMixed Phenotype Acute LeukemiaLymphoblastic Lymphoma
St. Jude Children's Research Hospital850 enrolled3 locationsNCT06289673