Updated daily · U.S. National Library of Medicine

Leukemia Clinical Trials

We surface trials from the U.S. National Library of Medicine with plain-English explanations of what each trial is actually testing. Whether you have newly diagnosed, relapsed, or treatment-resistant leukemia — knowing your options is the first step.

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Data sourced from the U.S. National Library of Medicine. Plain-English summaries are AI-generated to help you understand — always verify with your medical team.

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Understanding leukemia trial markers

Leukemia is defined by cytogenetics, molecular mutations, and disease subtype that determine prognosis and trial eligibility. Your bone marrow biopsy, flow cytometry, and genetic testing results are essential for matching to the right trials.

Cytogenetics & FISH
Essential classification
Chromosomal abnormalities (t(9;22), t(15;17), complex karyotype) define AML risk groups and predict chemotherapy response. FISH testing identifies specific translocations that predict trial eligibility.
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FLT3-ITD Mutation
~30% of AML
FLT3-ITD (internal tandem duplication) is adverse prognostic factor in AML. FLT3 inhibitors (midostaurin, sorafenib, crenolanib) are approved or in trials; ITD-positive status expands treatment options.
NPM1 & DNMT3A
~50-60% of AML
NPM1 and DNMT3A mutations without FLT3-ITD define favorable-risk AML. Venetoclax + azacitidine is standard for unfit patients. Trials test optimized combinations and durations.
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TP53 Mutation
~10% of AML, adverse
TP53 mutations associate with complex karyotype and very poor prognosis. Venetoclax combinations and novel targeted agents are being tested in TP53-mutant trials.
BCR-ABL Fusion (CML)
100% of CML
Philadelphia chromosome (t(9;22)) defines CML. Tyrosine kinase inhibitors (imatinib, dasatinib, nilotinib) are standard. Resistance mutations (T315I) predict need for second/third-generation TKIs or allogenic transplant.
Treatment History & Resistance
Defines relapsed status
Prior induction therapy response, time to relapse, and chemotherapy sensitivity guide second-line options. Early relapse (<6 months) vs late relapse (>12 months) impacts trial eligibility and prognosis.
Not sure which biomarkers you have? Upload your pathology or genomic report. We'll read it and match you to recruiting trials →

Common questions about lung cancer trials

Acute Leukemia (AML, ALL): Rapid onset, immature blast cells (>20% in bone marrow). Requires immediate treatment. AML median age ~68 years; ALL more common in children but can occur in adults. Without treatment, acute leukemia is rapidly fatal.

Chronic Leukemia (CML, CLL): Slower onset, mature cells elevated. Often discovered incidentally. CML and CLL can be managed with observation initially if asymptomatic. CLL median age ~70 years; CML can occur at any age.

Treatment urgency: Acute leukemia requires immediate therapy (days to weeks). Chronic leukemia can be monitored initially; treatment started when disease progresses or becomes symptomatic.

Knowing your leukemia subtype (AML vs ALL vs CML vs CLL) is the foundation for trial matching.

FLT3-ITD (internal tandem duplication) is a mutation in the FLT3 gene found in ~30% of AML patients. This mutation predicts:

Negative prognostic impact: FLT3-ITD positive AML is higher-risk and has worse prognosis with chemotherapy alone. Without targeted therapy, outcomes are poor.

Targeted therapy options: FLT3 inhibitors (midostaurin, sorafenib, crenolanib) added to chemotherapy improve outcomes. FLT3-ITD positive patients should ALWAYS receive FLT3 inhibitor therapy as part of frontline treatment.

Clinical trial implications: If FLT3-ITD positive, you likely qualify for trials testing FLT3 inhibitor combinations with chemotherapy or other agents. Many trials are specifically designed for FLT3-ITD AML.

Ask your hematologist about your FLT3 status immediately — it significantly impacts treatment selection and trial eligibility.

Newly Diagnosed Leukemia: First presentation of leukemia, no prior chemotherapy. Treatment goal: achieve complete remission (CR) — normal bone marrow with <5% blasts. Many trials focus on optimized first-line chemotherapy or targeted combinations (venetoclax + azacitidine for older AML patients).

Relapsed Leukemia: Return of leukemia after prior chemotherapy-induced remission. Relapse can be early (<6 months from end of chemotherapy) or late (>12 months). Early relapse is chemotherapy-resistant and requires alternative approaches (venetoclax combinations, targeted therapy, CAR-T, allogenic transplant).

Refractory Leukemia: Never achieved remission with initial chemotherapy. Very poor prognosis; requires salvage regimens (high-dose cytarabine, venetoclax, hypomethylating agents, clinical trials testing novel combinations).

Your disease status (newly diagnosed vs relapsed/refractory) dramatically changes trial options. Be clear about your prior treatments and response when enrolling.

If you progressed or relapsed after initial chemotherapy:

For Early Relapse AML (<6 months from remission):

  • Venetoclax + hypomethylating agents (azacitidine or decitabine)
  • Salvage high-dose cytarabine
  • FLT3 inhibitors (if FLT3-mutant)
  • IDH inhibitors (if IDH1/IDH2 mutant)
  • Allogenic stem cell transplant
  • CAR-T therapy (if eligible)
  • Clinical trials testing novel combinations

For CML Resistance:

  • Switch to higher-potency TKI (dasatinib, nilotinib if on imatinib)
  • Check BCR-ABL kinase domain mutations (T315I is imatinib-resistant)
  • T315I-specific inhibitors (ponatinib)
  • Allogenic transplant for blast crisis

For CLL Progression:

  • BTK inhibitors (ibrutinib, venetoclax, acalabrutinib)
  • Venice clax + rituximab combinations
  • CAR-T therapy (approved for relapsed/refractory CLL)
  • Allogenic transplant for chemotherapy-refractory disease

Molecular testing at relapse/progression can identify actionable mutations. Early discussion with your hematologist-oncologist about salvage options and clinical trials is essential.

Upload your pathology report and treatment history to find matching trials →

Yes, cytogenetics and molecular testing are essential for leukemia trial eligibility:

Standard cytogenetics (karyotype): Performed on bone marrow aspirate at diagnosis. Identifies chromosomal abnormalities that define risk groups and guide treatment intensity.

FISH testing: Rapid identification of specific translocations (t(9;22) in CML, t(15;17) in APL). Critical for prognosis and treatment decision-making.

Molecular mutations (FLT3, NPM1, DNMT3A, TP53, IDH1/IDH2): Increasingly tested at diagnosis in AML. Define risk stratification and predict response to targeted therapy. Essential for trial matching.

BCR-ABL kinase domain sequencing (CML): If resistance to TKI develops, mutation testing identifies which next-generation TKI to use.

Flow cytometry: Identifies blast phenotype (myeloid vs lymphoid), maturity status, and immunophenotypic abnormalities that define leukemia subtype.

Ask your hematologist about comprehensive testing — many trials require specific molecular profiles.

Gather these key documents before calling a trial:

(1) Bone marrow report: Biopsy and aspirate showing blast percentage, morphology, and lineage (AML vs ALL vs CML vs CLL).

(2) Cytogenetics & FISH: Karyotype results and FISH panel results showing all chromosomal abnormalities and translocations.

(3) Molecular testing: FLT3-ITD status, NPM1, DNMT3A, TP53, IDH1/IDH2, and any other sequencing results available.

(4) Flow cytometry report: Immunophenotyping results confirming disease lineage and maturity.

(5) Prior treatment history: Dates and names of all prior chemotherapy, targeted therapy (TKIs for CML), stem cell transplant if done. Response to each (CR, PR, no response).

(6) Blood work & performance status: Recent CBC, liver/kidney function, and ECOG performance status (0-3) from your oncologist.

(7) Current disease status: Latest blast percentage, WBC count, and LDH level. Are you in remission? If relapsed, when did relapse occur?

MyCancerTrialMatch generates a checklist specific to your matched trials to keep you organized.

Understanding clinical trials

What is a Phase 1, 2, or 3 trial?

Phase 1 tests safety. Phase 2 tests whether the treatment works. Phase 3 compares the new treatment against the current standard. Most patients look for Phase 2 or 3 trials, where the treatment has shown early promise.

What is an inclusion/exclusion criteria?

Every trial has a checklist of who can and cannot join. These might include your cancer stage, specific gene mutations, prior treatments, age, and overall health. Meeting these is required — not optional.

Do I have to pay for a clinical trial?

No. The experimental treatment in a clinical trial is free. You may still have to pay for routine care like doctor visits, but the trial drug or procedure itself is covered by the sponsor.

What does "recruiting" mean?

Recruiting means the trial is actively looking for participants right now. "Not yet recruiting" means it's approved but hasn't started. "Active, not recruiting" means it's ongoing but has all the participants it needs.