NPM1 mutation

Approved therapies

Approvals in the United States (FDA) and the European Union (EMA), one row per drug, regimen and tumour type. Where we have them, the pivotal trial's results and side effects are shown under each therapy, from the official label. Results from different trials are not directly comparable: patients, earlier treatments and comparators differ.

Drugs in active trials

Targeted drugs, antibody-drug conjugates and immunotherapies in active trials on this page, approved ones first, then by the furthest phase reached. Chemotherapy and supportive medicines are left out. "Not yet classified" means an early drug known only by its code.

16 of 16 drugs shown

Upcoming readouts

Active phase 2 and 3 trials whose primary completion date falls in the next 12 months: the date the main results are due to be collected, according to the sponsor. Results are usually published months later, and these dates often slip.

Companies

Industry sponsors leading active trials, by number of trials. Each square is one trial, shaded by phase.

1. Kura Oncology 4
2. Johnson & Johnson 3
3. Syndax Pharmaceuticals 3
4. Kyowa Kirin 1
5. AstraZeneca 1
6. Institut de Recherches Internationales Servier (I.R.I.S.) 1
7. Miltenyi Biomedicine 1
8. Servier 1
9. Sumitomo Pharma America 1
10. Charm Therapeutics 1
11. HUTCHMED 1

Phase 1 Phase 2 Phase 3 Phase 4 No phase

Trials

Matched automatically from registry text, so a few trials may only mention NPM1 mutation rather than require it. Check the quoted criterion, and the registry entry before relying on a match.

51 of 74 trials shown

1. Bleximenib in Combination With Standard Induction and Consolidation Therapy Followed by Maintenance for Treatment of Patients With Acute Myeloid Leukemia (AML)

   Recruiting Phase 3 Stichting Hemato-Oncologie voor Volwassenen Nederland Started 2026Primary completion 2030-06 NCT07223814

   bleximenibcytarabinedaunorubicinidarubicin

   New diagnosis of AML (≥10% blasts in BM or peripheral blood) with mutated NPM1 or with recurring rearrangements involving KMT2A according to ICC 2022 criteria.

   Acute Myeloid Leukemia

2. Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML

   Recruiting Phase 3 Kura Oncology Started 2025Primary completion 2031-11 NCT07007312

   ziftomenib FDAvenetoclaxazacitidinedaunorubicincytarabine

   …nintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia

   Acute Myeloid Leukemia (AML)

3. A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML)

   Recruiting Phase 3 Johnson & Johnson Started 2025Primary completion 2029-06 NCT06852222

   bleximenibvenetoclaxazacitidine

   …d lysine N-methyltransferase 2A gene rearranged (KMT2Ar) or nucleophosmin 1 gene mutated (NPM1m) acute myeloid leukemia (AML) with greater than or equal to (\> or =) 10% bone marrow bla…

   Leukemia, Myeloid, Acute

4. Revumenib in Combination With Azacitidine + Venetoclax in Patients NPM1-mutated or KMT2A-rearranged AML

   Recruiting Phase 3 Stichting Hemato-Oncologie voor Volwassenen Nederland Started 2025Primary completion 2029-12 NCT06652438

   revumenib FDA

   Patient with newly diagnosed NPM1-mutated AML, consistent with NPM1c, according to the 2022 International Consensus Classification …

   Acute Myeloid Leukemia, Adult

5. Study of Revumenib in Combination With Intensive Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia (AML) With a NPM1 Mutation

   Recruiting Phase 3 Syndax Pharmaceuticals Started 2025Primary completion 2029-06 NCT07211958

   revumenib FDAIntensive Chemotherapy Regimen

   Presence of an NPM1 mutation.

   Acute Myeloid Leukemias

6. Ziftomenib for the Treatment of Patients With NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia Not Eligible for Standard Therapy

   Recruiting Phase 2 Uma Borate Started 2026Primary completion 2027-12 NCT06930352

   cytarabineHydroxyureaziftomenib FDA

   KMT2A rearrangement (defined as KMT2A translocations) OR NPM1 mutation (defined as NPM1 mutation resulting in cytoplasmic localization, or NPM1c) OR other mutat…

   Acute Myeloid Leukemia

7. Study of AZD3632 Monotherapy or in Combination With Anticancer Agents in Participants With Advanced Haematologic Malignancies With KMT2Ar, NPM1m, or Other Genotypes Associated With HOX Overexpression

   Recruiting Phase 1/2 AstraZeneca Started 2026Primary completion 2029-02 NCT07155226

   AZD3632Posaconazole

   b) for Backfill - diagnosis of harbouring a KMT2Ar or NPM1m per local testing.

   Acute Lymphoblastic Leukaemia, Acute Myeloid Leukaemia, Higher-risk Myelodysplastic Syndromes

8. S243249 Monotherapy in Patients With Relapsed or Refractory Acute Leukemia With a KMT2A or NUP98 Translocation or Relapsed or Refractory AML With NPM1c Mutation

   Not yet recruiting Phase 1/2 Servier Started 2026Primary completion 2029-08 NCT07722312

   S243249 600mgS243249 400mgS243249 450mgS243249 300mg

   Confirmation of KMT2At, NUP98t, or NPM1c mutation using next generation sequencing (NGS), fluorescence in situ hybridization (FISH), or pol…

   Relapsed or Refractory Acute Leukemia, Relapsed or Refractory Acute Myeloid Leukemia

9. A Phase 2 Clinical Study of Ziftomenib in Patients With Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

   Recruiting Phase 2 Kyowa Kirin Started 2026Primary completion 2027-03 NCT07623616

   ziftomenib FDA

   A Phase 2 Clinical Study of Ziftomenib in Patients With Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

   Acute Myeloid Leukemia (AML)

10. A Study of Revumenib and Mezigdomide in People With Leukemia

    Recruiting Phase 1/2 Memorial Sloan Kettering Cancer Center Started 2026Primary completion 2029-01 NCT07356154

    revumenib FDAMezigdomide

    Participant has confirmed acute leukemia with detectable NPM1c, KMT2A translocation, or NUP98 translocation.

    Leukemia, Acute Leukemia, Relapse Leukemia, Refractory Leukemia

11. Study on the Efficacy and Safety of VA Regimen Compared to "3+7" Regimen in Newly Diagnosed AML With NPM1 or IDH1/IDH2 Mutations

    Not yet recruiting Phase 2 Shen yang Started 2026Primary completion 2028-05 NCT07664839

    azacitidinecytarabinedaunorubicin

    …e Efficacy and Safety of VA Regimen Compared to "3+7" Regimen in Newly Diagnosed AML With NPM1 or IDH1/IDH2 Mutations

    Acute Myeloid Leukemia, NPM1 Mutation, IDH1 Mutation, IDH2 Mutation

12. Menin-Inhibitor Targeted Maintenance in AML

    Not yet recruiting Phase 2 Center for International Blood and Marrow Transplant Research Started 2026Primary completion 2031-06 NCT07563010

    revumenib FDA

    NPM1-mutated (NPM1m) AML (Including FLT3-ITD or TKD co-mutation) 3.

    Relapsed Adult AML, Stem Cell Transplant Complications, Acute Myeloid Leukemia, Transplant-Related Hematologic Malignancy

13. MB-dNPM1-TCR.1 in Relapsed/Refractory AML

    Recruiting Phase 1/2 Miltenyi Biomedicine Started 2024Primary completion 2028-06 NCT06424340

    MB-dNPM1-TCR.1

    AML has NPM1 mutation which is recognized by dNPM1-TCR.1 and for which a specific Q-PCR is available for diseas…

    Leukemia, Myeloid, Acute

14. BN104 in Combination With Chemotherapy or Targeted Agents for Acute Myeloid Leukemia

    Recruiting Phase 1/2 Chen Suning Started 2024Primary completion 2025-12 NCT06746519

    venetoclaxazacitidine

    …gnosis according to the 2022 ELN Risk Stratification, as well as a defined combination of NPM1 mutation or NUP98 rearrangement or KMT2A rearrangement

    Acute Myeloid Leukemia

15. Venetoclax Plus Azacitidine Versus Intensive Chemotherapy for Fit Patients With Newly Diagnosed NPM1 Mutated AML

    Recruiting Phase 2 Technische Universität Dresden Started 2024Primary completion 2030-09 NCT05904106

    venetoclaxazacitidine

    Newly diagnosed CD33-positive AML with NPM1 mutation according to WHO criteria

    Acute Myeloid Leukemia

16. A Study of BN104 in the Treatment of Acute Leukemia

    Active, not recruiting Phase 1/2 Institut de Recherches Internationales Servier (I.R.I.S.) Started 2023Primary completion 2026-12 NCT06052813

    BN104 monotherapyBN104 monotherapy - rp2d

    Patients in the Phase II (single-arm pivotal clinical study) must have a confirmed NPM1 mutation or KMT2A rearrangement.

    ALL, Adult, AML, Adult, Acute Leukaemia, KMT2A Rearrangement

17. A Phase I-II Study Investigating the All-Oral Combination of the Menin Inhibitor SNDX-5613 With Decitabine/Cedazuridine (ASTX727) and Venetoclax in Acute Myeloid Leukemia (SAVE)

    Recruiting Phase 1/2 M.D. Anderson Cancer Center Started 2022Primary completion 2026-12 NCT05360160

    revumenib FDAvenetoclaxdecitabine

    …d/refractory (R/R cohort) AML or myeloid phenotype MPAL with either KMT2Ar, or NUP98r, or NPM1c.

    Acute Myeloid Leukemia

18. A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1)

    Recruiting Phase 1/2 Sumitomo Pharma America Started 2022Primary completion 2027-06 NCT04988555

    enzomenibazolesvenetoclaxgilteritinibazacitidineIntensive chemotherapy with 7 + 3

    …ired by regulatory authorities, participants must have a documented KMT2A (MLL) fusion or NPM1 mutation, including those with coexisting FLT3 genomic alterations and/or IDH1/2 mutation.

    Leukemia, Myeloid, Acute, Leukemia, Lymphocytic, Acute, Multiple Myeloma, Myelodysplastic Syndromes

19. A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)

    Recruiting Phase 1/2 Johnson & Johnson Started 2021Primary completion 2027-02 NCT04811560

    bleximenib

    AML harboring KMT2A-r (gene rearrangement/translocation) or NPM1 mutations only

    Acute Leukemia

20. A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation

    Recruiting Phase 1/2 Syndax Pharmaceuticals Started 2019Primary completion 2027-12 NCT04065399

    revumenib FDAcobicistat

    …Version 3.2020), or acute leukemia harboring KMT2A rearrangement, NUP98 rearrangement, or NPM1 mutation that have detectable disease in the bone marrow.

    Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Mixed Lineage Acute Leukemia, Mixed Phenotype Acute Leukemia

21. First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia

    Active, not recruiting Phase 1/2 Kura Oncology Started 2019Primary completion 2028-10 NCT04067336

    ziftomenib FDAmidazolamitraconazole

    Patients with a documented nucleophosmin 1 mutation (NPM1-m)

    Advanced Malignant Neoplasm, Acute Myeloid Leukemia, Mixed Lineage Leukemia, Mixed Lineage Acute Leukemia

22. Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy

    Active, not recruiting Phase 2 Masonic Cancer Center, University of Minnesota Started 2019Primary completion 2020-06 NCT03674411

    fludarabinecyclophosphamideTotal Body Irradiation (TBI)tacrolimusMycophenolate Mofetil (MMF)pegfilgrastimBusulfan (BU)melphalanMGTA 456 Infusion

    Normal karyotype with mutated NPM1 but FLT3-ITD wild type

    Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Biphenotypic/Undifferentiated Leukemia, Chronic Myelogenous Leukemia

23. Ziftomenib + Mezigdomide in Adolesc. and Adults w/ R/R AML

    Recruiting Phase 1 Massachusetts General Hospital Started 2026Primary completion 2027-07 NCT07355335

    ziftomenib FDAMezigdomide

    …mphoid Tumors (5th Edition) with documented KMT2A rearrangement or NPM1 cytoplasmic-type (NPM1c) mutation.

    KMT2A-rearranged, NPM1-mutant Refractory or Relapsed AML

24. A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML

    Recruiting Phase 1 Charm Therapeutics Started 2026Primary completion 2027-12 NCT07751991

    CHM-029

    Positive for NPM1 mutation, or KMT2A or NUP98 rearrangements

    AML (Acute Myeloid Leukemia)

25. BSB-2002 After Cyclophosphamide and Fludarabine for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With NPM1 Mutation

    Not yet recruiting Phase 1 City of Hope Medical Center Started 2026Primary completion 2027-04 NCT07583303

    cyclophosphamidefludarabineSingle Agent Therapy

    Positive for NPM1 mutation type A, D, G or H.

    Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia

26. Revumenib for the Treatment of Acute Leukemia in Patients Post-Allogeneic Stem Cell Transplant

    Recruiting Phase 1 City of Hope Medical Center Started 2024Primary completion 2028-04 NCT06575296

    revumenib FDA

    Confirmed NPM1m AML with at least one of the following additional characteristics

    Acute Leukemia, Acute Leukemia of Ambiguous Lineage, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia

27. A Study of Revumenib in Combination With Intensive Chemotherapy in Participants With Acute Myeloid Leukemias

    Active, not recruiting Phase 1 Syndax Pharmaceuticals Started 2024Primary completion 2027-02 NCT06226571

    revumenib FDAChemotherapy RegimenHiDAC

    KMT2Ar, NPM1m, or NUP98r mutations identified by local laboratory prior to the first dose of revumenib.

    Acute Myeloid Leukemias

28. SNDX-5613 and Gilteritinib for the Treatment of Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia and Concurrent MLL-Rearrangement or NPM1 Mutation

    Recruiting Phase 1 Uma Borate Started 2024Primary completion 2027-01 NCT06222580

    gilteritinibrevumenib FDA

    NPM1 mutation, MLL gene rearrangement and any other mutation that has proven HOXA-MEIS1 overexpression …

    Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With KMT2A Rearrangement, Acute Myeloid Leukemia With NPM1 Mutation, Recurrent Acute Myeloid Leukemia

29. A Clinical Study of HMPL-506 in Patients With Hematological Malignancies

    Recruiting Phase 1 HUTCHMED Started 2024Primary completion 2027-10 NCT06387082

    HMPL-506

    3. 1\) Dose escalation phase: patient with MLL-rearranged and/or NPM1-mutant relapsed/refractory AML or ALL (confirmed as per the 2022 World Health Organization (WHO)…

    Hematological Malignancies

30. Revumenib in Combination With 7+3 + Midostaurin in AML

    Recruiting Phase 1 Richard Stone, MD Started 2024Primary completion 2027-03 NCT06313437

    revumenib FDAmidostaurincytarabinedaunorubicin

    Presence of FLT3-ITD and/or TKD mutation(s) AND NPM1 mutation in bone marrow or peripheral blood

    Acute Myeloid Leukemia, AML, Adult, AML With Gene Mutations, AML

31. Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia

    Recruiting Phase 1 St. Jude Children's Research Hospital Started 2024Primary completion 2027-01 NCT06177067

    revumenib FDAvenetoclaxazacitidinemethotrexatecytarabine

    Presence of KMT2A rearrangement (KMT2Ar), NUP98 rearrangement (NUP98r), NPM1 mutation or fusion, PICALM::MLLT10, DEK::NUP214, UBTF-TD, KAT6A rearrangement (KAT6Ar), or SET::NU…

    Refractory Acute Myeloid Leukemia, Relapsed Acute Myeloid Leukemia, Acute Leukemia of Ambiguous Lineage

32. Safety and Tolerability of Ziftomenib Combinations in Patients With Relapsed/Refractory Acute Myeloid Leukemia

    Recruiting Phase 1 Kura Oncology Started 2024Primary completion 2026-12 NCT06001788

    ziftomenib FDAfludarabineidarubicincytarabinegilteritinibGranulocyte colony-stimulating factor

    Has a documented NPM1 mutation or KMT2A rearrangement.

    AML, AML With Mutated NPM1, Hematologic Malignancy, KMT2Ar

33. A Phase 1b Study of Menin Inhibitor SNDX- 5613 in Combination With Daunorubicin and Cytarabine in Newly Diagnosed Patients With Acute Myeloid Leukemia and NPM1 Mutated/FLT3 Wildtype or MLL/KMT2A Rearranged or NUP98 Alterations Disease

    Recruiting Phase 1 National Cancer Institute (NCI) Started 2024Primary completion 2027-12 NCT05886049

    cytarabinedaunorubicinrevumenib FDA

    Dose escalation: Patients ages 18-75 years at time of diagnosis with NPM1-mutated/FLT3-ITD wildtype and NPM1-mutated/FLT3-TKD wildtype with high-risk features (adverse ris…

    Acute Myeloid Leukemia, Acute Myeloid Leukemia With KMT2A Rearrangement, Acute Myeloid Leukemia With NPM1 Mutation, Secondary Acute Myeloid Leukemia

34. A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML

    Recruiting Phase 1 Kura Oncology Started 2023Primary completion 2030-04 NCT05735184

    ziftomenib FDAvenetoclaxazacitidinedaunorubicincytarabinequizartinib

    Patients must have a documented NPM1 mutation or KMT2A rearrangement and have either newly diagnosed or relapsed/refractory AML

    Acute Myeloid Leukemia, Mixed Lineage Leukemia Gene Mutation, Refractory AML, AML With Mutated NPM1

35. A Study of Bleximenib in Combination With Acute Myeloid Leukemia (AML) Directed Therapies

    Active, not recruiting Phase 1 Johnson & Johnson Started 2022Primary completion 2026-10 NCT05453903

    bleximenibvenetoclaxazacitidinecytarabinedaunorubicinidarubicin

    c) harboring KMT2A, NPM1, NUP98, or NUP214 alterations

    Leukemia, Myeloid, Acute

36. XP-005 Personalized Vaccine Alone or in Combination With Toripalimab for the Prevention of Relapse After Remission in Acute Myeloid Leukemia

    Recruiting Early Phase 1 Shanghai Jiao Tong University School of Medicine Started 2025Primary completion 2027-01 NCT06980155

    Personalized neoantigen tumor vaccinePD-1 inhibitor

    NPM1 mutation classification as Type A, D, G, H, B, and J

    Acute Myeloid Leukemia

37. UPDATE AML: UPdated Disease Monitoring And Treatment for Enhanced Outcomes for Pediatric AML

    Recruiting Early Phase 1 Baylor College of Medicine Started 2025Primary completion 2028-10 NCT07059975

    idarubicinfludarabinecytarabinevenetoclaxetoposideAsparaginase Erwinia Chrysanthemi (recombinant)Intrathecal triple

    Mutated NPM1

    Acute Myeloid Leukemia, Pediatric AML

38. Study to Improve OS in 18 to 60 Year-old Patients, Comparing Daunorubicin Versus High Dose Idarubicin Induction Regimens, High Dose Versus Intermediate Dose Cytarabine Consolidation Regimens, and Standard Versus MMF Prophylaxis of GvHD in Allografted Patients in First CR

    Recruiting Phase 2/3 University Hospital, Angers Started 2015Primary completion 2025-07 NCT02416388

    idarubicindaunorubicincytarabineCyclosporinemethotrexateMycophenolic acid (MPA)vosaroxindexamethasonevenetoclax

    either initially favorable but poor molecular responders for NPM1 MRD: NPM1 mutation, without FLT3-ITD mutation or with an FLT3-ITD ratio \< 0.50 and MRD2 positive blood (dec…

    Broad match: NPM1 mutationAcute Myeloid Leukemia (AML)

39. Phase 2 Trial of BN104 as Post-HSCT Maintenance in Acute Leukemia

    Recruiting Phase 2 The First Affiliated Hospital of Soochow University Started 2025Primary completion 2027-12 NCT07101497

    BN104 monotherapy

    Harboring one of the following genetic aberrations: a. somatic NPM1 mutation (without FLT3-ITD/TKD co-mutations)

    Broad match: NPM1 mutationMenin Inhibitors, Post Hematopoietic Stem Cell Transplantation, Maintenance Therapy, Acute Leukemia

40. A Study of Azacitidine and Venetoclax in People With Acute Myeloid Leukemia (AML)

    Recruiting Phase 2 Memorial Sloan Kettering Cancer Center Started 2025Primary completion 2027-01 NCT06773208

    azacitidinevenetoclax

    …disease (MRD) at or above a level of 0.1%, by flow cytometry (MFC) or in molecular cases (NPM1 mutated or one of the CBF translocations) RT-qPCR at or above 0.01%, as described above (see sect…

    Broad match: NPM1 mutationAcute Myeloid Leukemia (AML)

41. Venetoclax + Azacitidine in Patients With Acute Myeloid Leukemia

    Active, not recruiting Phase 2 Grupo Cooperativo de Estudio y Tratamiento de las Leucemias Agudas y Mielodisplasias Started 2024Primary completion 2028-06 NCT06668558

    azacitidinevenetoclax

    In patients with NPM1 mutation, qRT-PCR of NPM1 will be the method used to establish a molecular failure, defined as fai…

    Broad match: NPM1 mutationAcute Myeloid Leukemia

42. Comparing Cytarabine + Daunorubicin Therapy Versus Cytarabine + Daunorubicin + Venetoclax Versus Venetoclax + Azacitidine in Younger Patients With Intermediate Risk AML (A MyeloMATCH Treatment Trial)

    Recruiting Phase 2 National Cancer Institute (NCI) Started 2024Primary completion 2027-12 NCT05554393

    azacitidinecytarabinedaunorubicinvenetoclax

    NPM1 mutation

    Broad match: NPM1 mutationAcute Myeloid Leukemia

43. Interferon-γ (IFN-γ) With Donor Leukocyte Infusion to Treat Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes Post Allogeneic Hematopoietic Stem Cell Transplantation

    Recruiting Phase 2 Sawa Ito, MD Started 2024Primary completion 2029-10 NCT06529731

    Interferon gamma-1bDonor Leukocyte Infusion (DLI)

    For patients with mutant NPM1, at least 1,000 mutant transcript copies per 106 ABL or equivalent housekeeping transcrip…

    Broad match: NPM1 mutationAcute Myeloid Leukemia, Myelodysplastic Syndromes

44. Allo HSCT Using RIC and PTCy for Hematological Diseases

    Recruiting Phase 2 Masonic Cancer Center, University of Minnesota Started 2023Primary completion 2027-10 NCT05805605

    Peripheral Blood Stem Cell TransplantAllopurinol 300 MGfludarabinecyclophosphamideBone Marrow Cell TransplantSirolimus PillMycophenolate Mofetil

    Normal karyotype with mutated NPM1 and wild type FLT-ITD (unless persistently NPM1 positive by PCR following two cycles of c…

    Broad match: NPM1 mutationAcute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Biphenotypic Acute Leukemia, Undifferentiated Leukemia

45. A Randomized Phase II Study of Hyperbaric Oxygen in Improving Engraftment in Umbilical Cord Blood Stem Cell Transplant

    Active, not recruiting Phase 2 University of Rochester Started 2019Primary completion 2027-06 NCT03739502

    Hyperbaric oxygen

    … as patients with t(15;17)(q22;q21), t(8;21)(q22;q22), inv(16)(p13q22)/t(16;16)(p13;q22), NPM1 mutation without FLT3-ITD, and double-mutated CEBPA58,59), AML in CR2 or subsequent CR, high-risk …

    Broad match: NPM1 mutationAML, NHL, Hodgkin Disease, All

46. Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders

    Recruiting Phase 2 Masonic Cancer Center, University of Minnesota Started 2018Primary completion 2027-06 NCT03314974

    HSCT with TBI RegimenHSCT with Non-TBI Regimen

    Normal karyotype with mutated NPM1 and wild type FLT-ITD

    Broad match: NPM1 mutationAcute Leukemia, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Lymphoma

47. 225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody With Fludarabine, Melphalan and Total Marrow and Lymphoid Irradiation as Conditioning Treatment for Donor Stem Cell Transplant in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome

    Recruiting Phase 1 City of Hope Medical Center Started 2025Primary completion 2028-05 NCT06287944

    Actinium Ac 225-DOTA-DaratumumabDaratumumabfludarabineIndium In 111-DOTA-DaratumumabmelphalanSirolimustacrolimus

    …ated abnormalities), or all patient in intermediate risk groups accept patients with FLT3-NPM1+ disease, OR

    Broad match: NPM1 mutationAcute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Myelodysplastic Syndrome

48. A Phase I Study Investigating the Combination of the Menin Inhibitor Ziftomenib With Venetoclax and Gemtuzumab in Pediatric Patients With Acute Myeloid Leukemia

    Recruiting Phase 1 M.D. Anderson Cancer Center Started 2025Primary completion 2028-12 NCT06448013

    venetoclaxgemtuzumab ozogamicinziftomenib FDA

    Presence of KMT2Ar, NUP98r, NPM1c, UBTF-ITD or other HOX pathway mutation.

    Broad match: NPM1 mutationAcute Myeloid Leukemia

49. Ziftomenib Maintenance Post Allo-HCT

    Recruiting Phase 1 Massachusetts General Hospital Started 2024Primary completion 2027-04 NCT06440135

    ziftomenib FDA

    NPM1 mutation

    Broad match: NPM1 mutationAcute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, NPM1 Mutation, KMT2A Rearrangement

50. 90Y-DOTA-anti-CD25 Basiliximab, Fludarabine, Melphalan, and Total Marrow and Lymphoid Irradiation for the Treatment of High-Risk Acute Leukemia or Myelodysplastic Syndrome

    Active, not recruiting Phase 1 City of Hope Medical Center Started 2022Primary completion 2027-06 NCT05139004

    BasiliximabfludarabineIndium In 111-DOTA-BasiliximabmelphalanPaliferminYttrium Y 90 Basiliximab

    …ated abnormalities), or all patient in intermediate risk groups accept patients with FLT3-NPM1+ disease

    Broad match: NPM1 mutationAcute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Myelodysplastic Syndrome, Secondary Acute Myeloid Leukemia

51. Haplo-Cord HSCT for AML/MDS

    Recruiting No phase Fujian Medical University Union Hospital Started 2025Primary completion 2028-10 NCT07588360

    fludarabinecytarabinemelphalancyclophosphamide

    Persistently positive MRD after chemotherapy in patients with NPM1-mutated AML.

    Broad match: NPM1 mutationMDS (Myelodysplastic Syndrome), AML (Acute Myeloid Leukemia)

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