Leukemia Clinical Trial Monitor — AML, ALL, CLL & CML Programs

Leukemia clinical trials in 2026: a rapidly evolving landscape

Leukemia encompasses several distinct diseases — AML, ALL, CLL, CML — each with its own trial ecosystem. The common thread in 2025–2026 is the rapid proliferation of targeted combination strategies, bispecific antibodies, and next-generation CAR-T approaches that are generating dozens of new Phase 1 and Phase 2 trials each quarter.

For hematology-focused pharma, biotech, and research teams, manually tracking new trials across all leukemia subtypes on ClinicalTrials.gov is increasingly impractical. A daily automated digest cuts through the noise.

Acute myeloid leukemia (AML): targeted therapy combinations

AML is generating the most trial volume among leukemia subtypes. Key areas of activity include:

FLT3 inhibitors: second and third generation

Midostaurin and gilteritinib established FLT3 inhibition as standard therapy. Current trials focus on next-generation inhibitors with improved selectivity, combinations with BCL-2 inhibitors, and approaches to overcome FLT3-ITD resistance after gilteritinib.

IDH1/IDH2 inhibitors

Ivosidenib and enasidenib demonstrated single-agent activity; ongoing trials are testing combinations with venetoclax, hypomethylating agents, and intensive chemotherapy backbones in newly diagnosed and relapsed/refractory settings.

BCL-2 inhibitor combinations

Venetoclax plus azacitidine has become a standard frontline regimen for unfit patients. The new wave of trials is testing venetoclax with targeted agents (FLT3i, IDH1/2i, menin inhibitors) and exploring how to sequence these combinations optimally.

Menin inhibitors

Revumenib and ziftomenib represent a new targeted class showing activity in NPM1-mutated and KMT2A-rearranged AML. Multiple Phase 1/2 trials are actively enrolling for combination strategies with standard regimens.

Acute lymphoblastic leukemia (ALL): bispecifics and CAR-T

The bispecific antibody blinatumomab (CD3×CD19) and CAR-T therapies have transformed relapsed/refractory B-cell ALL. The 2025–2026 trial landscape extends these approaches:

• Next-generation CD19/CD22 bispecific antibodies with improved half-life and tolerability
• CAR-T in earlier-line settings (post-frontline relapse rather than third-line)
• T-cell ALL: CAR-T programs targeting CD7, CD1a, and other T-ALL antigens
• Philadelphia chromosome-positive ALL: ponatinib combinations with blinatumomab
• MRD-directed therapy: using minimal residual disease to guide treatment intensification or de-escalation

Chronic lymphocytic leukemia (CLL): BTK inhibitor evolution

CLL treatment has been transformed by BTK inhibitors (ibrutinib, acalabrutinib, zanubrutinib). The current trial landscape focuses on what comes after:

• Non-covalent BTK inhibitors (pirtobrutinib) for C481S-mutated BTK-resistant disease
• BTK PROTAC degraders in development for broader resistance coverage
• Fixed-duration venetoclax + BTK inhibitor combinations for treatment-naive patients
• CAR-T and bispecific antibodies for double-refractory BTKi + BCL-2i disease
• Richter's transformation: specific programs for CLL transformation to aggressive lymphoma

Chronic myeloid leukemia (CML): TKI optimization

CML is largely managed with TKI therapy, but active trial areas include deep molecular response optimization for treatment-free remission eligibility, third-generation TKIs (asciminib) in earlier lines, and approaches to the rare BCR-ABL T315I mutation.

Who monitors leukemia clinical trials?

• Hematology-oncology pharma teams at companies with AML, ALL, or CLL programs tracking competitor trial activity
• Clinical development leaders designing new combination trials needing current competitive landscape data
• Medical science liaisons and medical affairs needing current knowledge of enrollment status and new trial openings
• Healthcare investors covering hematology-focused biotech companies
• Academic hematologists who refer patients to relevant trials and need to know what's enrolling nearby

Related hematology clinical trial monitors

• Multiple myeloma clinical trials — overlapping CAR-T, bispecific, and proteasome inhibitor programs
• Rare disease clinical trials — including rare leukemia variants and MDS programs
• Breast cancer clinical trials — for teams tracking CDK4/6 and other programs across tumor types