Updated daily · U.S. National Library of Medicine

Leukemia Clinical Trials

We surface recruiting trials for AML, CML, ALL, and CLL from ClinicalTrials.gov. Your specific mutation — FLT3, IDH1/2, NPM1, BCR-ABL, or CD19 — determines which trials you qualify for and which to prioritize.

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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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Key biomarkers for leukemia trials

Leukemia trial eligibility is driven by your specific mutation profile. These are the most actionable biomarkers across AML, CML, ALL, and CLL — find them on your bone marrow biopsy or molecular panel report.

FLT3-ITD / FLT3-TKD
~30% of AML
Most actionable mutation in AML. Targeted by midostaurin, gilteritinib, quizartinib. Many Phase 2/3 trials recruiting — especially for relapsed/refractory and post-transplant settings.
Find FLT3 trials →
NPM1 / Menin pathway
~30% of AML
NPM1-mutant AML responds well to menin inhibitors (revumenib, ziftomenib). Multiple Phase 2/3 trials actively recruiting — this is one of the fastest-growing trial categories in AML.
Find NPM1/Menin trials →
IDH1 / IDH2
~20% of AML
Targeted by ivosidenib (IDH1) and enasidenib (IDH2). Combination trials with venetoclax + azacitidine are actively recruiting in frontline and relapsed settings.
Find IDH trials →
BCR-ABL (Philadelphia+)
Defines CML; ~25% of adult ALL
TKI resistance in CML — especially T315I mutation — drives trial eligibility. Ponatinib and asciminib (STAMP inhibitor) trials available. Ph+ ALL has specific CAR-T and TKI combination trials.
Find BCR-ABL trials →
CD19 / CAR-T eligibility
Key target in ALL and CLL
CD19-targeted CAR-T (tisagenlecleucel, brexucabtagene) is approved for relapsed ALL. Trials are expanding to first-line settings, CD19+CD22 dual targeting, and CLL at academic centers.
Find CAR-T trials →
TP53 mutation
~10% of AML; ~10% of CLL (17p del)
Associated with poor prognosis and resistance to standard therapy. Trials testing eprenetapopt (APR-246), magrolimab (anti-CD47), and venetoclax combinations specifically for TP53-mutant disease.
Find TP53 trials →
Not sure which mutations you have? Upload your bone marrow biopsy or molecular panel report. We'll read it and match you to recruiting trials →

Common questions about leukemia trials

FLT3-ITD is found in ~30% of AML patients and is one of the most actionable mutations. Approved agents: midostaurin (Rydapt) and gilteritinib (Xospata). Active trials include quizartinib combinations, next-generation FLT3 inhibitors in frontline settings, and post-transplant maintenance trials.

Confirm your FLT3 subtype (ITD vs TKD) — both qualify for trials but with different response profiles.

Imatinib resistance is usually caused by BCR-ABL kinase domain mutations. The first step is testing for T315I mutation — it resists most TKIs but responds to ponatinib (Iclusig) and asciminib (Scemblix). Trials are available for ponatinib combinations, novel allosteric inhibitors, and CML in blast crisis.

Upload your mutation testing results to see which trials apply →

CAR-T therapy engineers your own T-cells to attack cancer. For ALL, CD19-targeted CAR-T (tisagenlecleucel, brexucabtagene) is approved for relapsed/refractory disease. Trials are expanding to first-line, CD22 targets, and dual-target constructs.

Key eligibility factors: CD19 expression level, number of prior treatment lines, performance status, and organ function. CLL patients may also qualify at academic centers.

IDH mutations affect ~20% of AML patients. Approved: enasidenib for IDH2, ivosidenib for IDH1. Active trials include IDH inhibitor + venetoclax + azacitidine combinations, frontline settings, and novel agents like olutasidenib.

IDH mutation testing should be part of your initial AML workup — ask your hematologist if it was performed.

Yes. Post-transplant relapse trial options include: donor lymphocyte infusion (DLI) combinations, second CAR-T infusions, bispecific antibody trials (flotetuzumab, CD3xCD123), and novel targeted agents. The speed of relapse relative to transplant date matters for eligibility.

Several academic centers have specific post-transplant relapse protocols. Upload your transplant records to match accurately.

Leukemia trials often enroll faster than solid tumor trials. The fastest path: (1) Ask your hematologist if your center participates in NCI cooperative group trials (ECOG-ACRIN, SWOG, Alliance). (2) Contact the coordinator the same day with your mutation profile and treatment history. (3) Pre-screening calls can often happen within 24–48 hours.

Alongside provides a same-day contact script so you don't waste time on your first call.

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.