Leukemia clinical trials: a rapidly evolving landscape
Leukemia encompasses four major disease subtypes, AML, ALL, CLL, CML, each with a distinct trial ecosystem. The live trial data on this page shows the current count of active leukemia trials on ClinicalTrials.gov, 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.
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Sign Up FreeA primary completion date pushed out, enrollment cut, or a primary endpoint changed in the last 90 days. Active trials only.
| Trial | Warning signs | Status |
|---|---|---|
| CD34+ Transplants for Myeloid Malignancies NCT05565105 | Primary completion moved at least 6 months later Primary endpoint(s) modified Enrollment reduced 50% | Not Yet Recruiting |
180 more on the Trials at Risk page. See which trials →
Phase 2 and Phase 3 trials with a primary completion date in the next 180 days, as ClinicalTrials.gov estimates it today. Active trials only.
| Trial | Scheduled | Phase |
|---|---|---|
| Venetoclax in Patients With MDS or AML in Relapse After AHSCT NCT05226455 | Nov 15, 2026 · Estimated | Phase 1/2 |
186 more on the Completions page. See the calendar →
Acute myeloid leukemia (AML): targeted therapy combinations
AML carries the highest trial volume of any leukemia subtype, with Phase 3 programs enrolling or in follow-up alongside a deep early-phase pipeline. 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 carries a broad active pipeline with multiple 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 active, not 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 carries a deep Phase 3 pipeline, 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 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
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2468 active trials, 1426 recruiting.
Industry sponsors run 477 of these trials. The rest are run by universities, hospitals, government bodies and other non-commercial sponsors. The table below lists the 5 largest of 225.
| Top industry sponsor | Active trials |
|---|---|
| Novartis Pharmaceuticals | 27 |
| AbbVie | 21 |
| AstraZeneca | 21 |
| Ascentage Pharma Group Inc. | 13 |
| BeOne Medicines | 12 |
| Trial | Status | Latest tracked change | Seen |
|---|---|---|---|
| BLAST MRD AML-2: BLockade of PD-1 Added to Standard Therapy to Target… NCT04284787 | Active, Not Recruiting | Study completion pushed from Jan 30, 2027 to Oct 9, 2027 | 2026-11-14 |
| Total Marrow and Lymphoid Irradiation, Fludarabine, and Melphalan Before Donor… NCT03494569 | Active, Not Recruiting | Enrollment increased: 36 → 54 participants | 2026-11-14 |
| Anti-CD7 CAR-T Cells in Relapsed/Refractory T-Cell Acute Lymphoblastic Leukemia… NCT06934382 | Active, Not Recruiting | Enrollment closed, study ongoing | 2026-11-14 |
| BLAST MRD AML-1: BLockade of PD-1 Added to Standard Therapy to Target… NCT04214249 | Active, Not Recruiting | Study completion pushed from Jan 30, 2027 to Oct 9, 2027 | 2026-11-14 |
| A Clinical Trial of MK-1045 in People With B-cell Cancer (MK-1045-006) NCT07709000 | Recruiting | Trial sites expanded: 7 → 10 locations | 2026-11-14 |
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