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

Leukemia clinical trials in 2026: a rapidly evolving landscape

Leukemia encompasses four major disease subtypes — AML, ALL, CLL, CML — each with a distinct trial ecosystem. As of March 2026, there are 636 active leukemia trials on ClinicalTrials.gov (876 total registered), spanning Phase 1 through Phase 3 across all subtypes. The common thread in 2025–2026 is the rapid proliferation of targeted combination strategies, bispecific antibodies, and next-generation CAR-T approaches generating dozens of new trials each quarter.

Subtype	Active Trials	Phase 3 Active	Primary Drug Classes
AML	242	17	FLT3i, IDH1/2i, venetoclax combos, menin inhibitors
ALL	151	14	Blinatumomab, CAR-T (CD19/CD22), inotuzumab
CLL	143	22	BTK inhibitors, venetoclax, BTK degraders (PROTACs)
CML	32	3	Asciminib (STAMP inhibitor), TKI discontinuation, TFR

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 has 242 active trials — the highest volume of any leukemia subtype — with 17 Phase 3 programs currently enrolling or in follow-up. The defining feature of the 2025–2026 AML landscape is the convergence of multiple targeted agents that can now be combined: FLT3 inhibitors, IDH inhibitors, venetoclax, and menin inhibitors are each approved or late-stage, and the field is now studying how to sequence and combine them to deepen remissions and overcome resistance. See the full AML trial tracker →

Menin inhibitors: the newest approved class

Revumenib (Syndax) became the first menin inhibitor approved by the FDA in 2024 for relapsed/refractory AML with KMT2A rearrangement. Ziftomenib (Kura Oncology) is in active Phase 3 development (NCT07007312) testing combinations with venetoclax+azacitidine or 7+3 induction chemotherapy in NPM1-mutant and KMT2A-rearranged AML. The menin-KMT2A-MLL axis represents the most clinically validated new dependency in AML discovered in the past five years.

FLT3 inhibitors: MRD-guided maintenance

Midostaurin and gilteritinib established FLT3 inhibition as standard therapy. The frontier in 2026 is MRD-guided maintenance after allogeneic transplant — a Phase 3 trial (NCT07463651) is directly comparing gilteritinib vs. sorafenib in FLT3+ patients based on post-transplant MRD status. Next-generation FLT3 inhibitors with improved selectivity and CNS penetration are advancing in Phase 1/2.

BCL-2 inhibitor combinations

Venetoclax plus azacitidine is standard frontline therapy for unfit patients. The current Phase 3 wave (NCT04628026, NCT07396480) tests whether adding venetoclax to standard induction and transplant conditioning improves outcomes in fit patients. The parallel question is how to sequence or combine venetoclax with FLT3i, IDH1/2i, and menin inhibitors in patients who are simultaneously eligible for multiple targeted agents.

IDH1/IDH2 inhibitors

Ivosidenib and enasidenib established single-agent activity in IDH-mutant R/R AML. Ongoing trials test these agents combined with venetoclax, azacitidine, and intensive chemotherapy in newly diagnosed and R/R settings. An oral azacitidine prodrug (cedazuridine/azacitidine; ASTX030, NCT04256317, Phase 3) is actively enrolling in myeloid neoplasms and may reshape how azacitidine-backbone regimens are delivered.

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

ALL has 151 active trials and 14 Phase 3 programs. The bispecific antibody blinatumomab (CD3×CD19) and CAR-T therapies have transformed relapsed/refractory B-cell ALL — the 2025–2026 trial landscape is now moving these proven approaches earlier in the treatment sequence and into pediatric settings. See the full ALL trial tracker →

• Blinatumomab in frontline therapy: NCT03914625 (NCI Phase 3) testing blinatumomab combined with standard chemotherapy in newly diagnosed B-ALL — results pending with potential to reshape induction protocols
• Inotuzumab ozogamicin earlier: NCT03959085 (Children's Oncology Group Phase 3) adding inotuzumab to post-induction chemotherapy in pediatric B-ALL with high MRD after induction
• CAR-T in earlier lines: Programs now enrolling in post-frontline relapse (rather than third-line only), with MRD-positive patients being studied as a bridge-to-transplant or transplant-replacement strategy
• T-cell ALL: CAR-T programs targeting CD7, CD1a, and other T-ALL antigens in Phase 1/2 — the frontline remains chemotherapy-intensive but targeted approaches are advancing
• Philadelphia chromosome-positive ALL: Ponatinib/blinatumomab combinations (NCT04530565, NCI Phase 3) now actively recruiting for Ph+ ALL
• MRD-directed therapy: Multiple Phase 3 trials use MRD as a guide to intensify or de-escalate — the goal is curing more patients while reducing long-term chemotherapy toxicity

Chronic lymphocytic leukemia (CLL): BTK inhibitor evolution

CLL has the highest Phase 3 trial count of any leukemia subtype — 22 active Phase 3 programs — reflecting an extremely active post-BTKi drug development environment. BTK inhibitors (ibrutinib, acalabrutinib, zanubrutinib) transformed CLL; the current question is what happens when those agents stop working. See the full CLL trial tracker → | BTK inhibitor pipeline overview →

• Non-covalent BTK inhibitors: Pirtobrutinib (Lilly) is approved for C481S-mutated BTK-resistant disease; nemtabrutinib (Merck) is in Phase 3 trials — both bind the BTK active site without forming a covalent bond, bypassing the primary resistance mechanism
• BTK degraders (PROTACs): BGB-16673 (BeiGene) has 5 Phase 3 trials currently enrolling — the first BTK PROTAC in late-stage development, designed to degrade rather than just inhibit BTK and overcome both C481S and alternative resistance mutations
• Fixed-duration venetoclax + BTKi: Multiple Phase 3 trials testing time-limited combination regimens in treatment-naive CLL — the goal is durable MRD-negative remissions without indefinite therapy
• CAR-T and bispecifics for double-refractory disease: Patients who have failed both BTK inhibitors and venetoclax represent a high unmet need; CD19/CD20 CAR-T and bispecific antibodies (anti-CD20×CD3) are entering Phase 2/3 for this population
• MRD-guided treatment-free remission: Landmark trials showing that venetoclax-based regimens can achieve MRD-negativity and support treatment discontinuation are spawning a new generation of fixed-duration combination trials

Chronic myeloid leukemia (CML): TKI optimization

CML has 32 active trials and 3 Phase 3 programs — the smallest trial volume of the four major subtypes, reflecting a disease that is now largely managed with standard TKI therapy (imatinib, dasatinib, nilotinib, bosutinib). Active trial areas include: deep molecular response optimization for treatment-free remission (TFR) eligibility, asciminib (STAMP inhibitor targeting the myristoyl pocket) now approved and moving into earlier-line testing, ponatinib combinations for T315I gatekeeper mutations, and approaches to CML blast crisis which has outcomes similar to de novo AML.

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