6 recruiting, 4 unknown, 2 active not recruiting, 2 completed. 11 Phase 1, 8 Phase 2, 1 Phase 3.
Follow GATA2 Deficiency: new trials by email Literature Review of GATA2 Deficiency trials Research Report on GATA2 Deficiency
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study to Evaluate S241656 Alone or in Combination in Participants With Selected Myeloid Malignancies S241656, Posaconazole - Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia | Phase 1 / Phase 2 | Not yet recruiting | Servier | 64 | 2026-09-15 | 2026-07-22 |
| Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes Biospecimen Collection, Bone Marrow Aspiration, Cirtuvivint - Acute Myeloid Leukemia, Myelodysplastic Syndrome | Phase 1 | Recruiting | National Cancer Institute (NCI) | 54 | 2025-08-15 | 2026-11-03 |
| Regorafenib in Combination With Venetoclax and Azacitidine for the Treatment of Patients With Relapsed or Refractory Acute Myeloid Leukemia Azacitidine, Biospecimen Collection, Bone Marrow Aspiration - Myelodysplastic Syndrome/Acute Myeloid Leukemia, Recurrent Acute Myeloid Leukemia | Phase 1 | Suspended | City of Hope Medical Center | 20 | 2025-04-25 | 2026-10-20 |
| Iadademstat in Combination With Azacitidine and Venetoclax in Treating Newly Diagnosed Acute Myeloid Leukemia Azacitidine, Biospecimen Collection, Bone Marrow Biopsy - Acute Myeloid Leukemia, Myelodysplastic Syndrome/Acute Myeloid Leukemia | Phase 1 | Recruiting | OHSU Knight Cancer Institute | 30 | 2024-08-22 | 2026-06-30 |
| A Study to Investigate APL-4098 Alone and in Combination in Adults With AML or MDS APL-4098, Azacitidine and APL-4098, Azacitidine and Venetoclax and APL-4098 - Acute Myeloid Leukemia Refractory, Myelodysplastic Syndrome Acute Myeloid Leukemia | Phase 1 | Recruiting | Apollo Therapeutics Ltd | 100 | 2024-06-04 | 2026-10-28 |
| A Study of QLF32101 in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndrome QLF32101 - Acute Myeloid Leukemia and Myelodysplastic Syndrome | Phase 1 | Unknown | Qilu Pharmaceutical Co., Ltd. | 122 | 2023-02-01 | 2023-01-31 |
| AB8939 in Patients With Relapsed/Refractory Acute Myeloid Leukemia AB8939, Venetoclax - Acute Myeloid Leukemia Refractory, Acute Myeloid Leukemia, in Relapse | Phase 1 / Phase 2 | Recruiting | AB Science | 78 | 2022-06-01 | 2025-10-02 |
| Total Lymphoid Irradiation Pre-HSCT in Severe Congenital Neutropenia conditioning with TLI - Severe Congenital Neutropenia, GATA2 Deficiency | Phase 2 | Unknown | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | 10 | 2021-04-14 | 2021-04-14 |
| A Phase I, Open-Label, Dose Escalation and Cohort Expansion Study of BS HH 002.SA in Patients With AML and MDS BS HH 002.SA - Acute Myeloid Leukemia, Myelodysplastic Syndrome | Phase 1 | Unknown | Shanghai Bensen Pharmaceutical Co., Ltd. | 66 | 2021-02-01 | 2021-02-08 |
| Nivolumab for High-Risk MDS/AML Patients After Allogeneic Stem Cell Transplant With Post-Transplant Cyclophosphamide Nivolumab - Leukemia, Myeloid, Acute, Myelodysplastic Syndromes | Phase 1 | Withdrawn | SCRI Development Innovations, LLC | 2020-04-13 | 2023-12-05 | |
| Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant Total-Body Irradiation, Thiotepa, Fludarabine - Acute Biphenotypic Leukemia, Acute Leukemia | Phase 2 | Active not recruiting | Fred Hutchinson Cancer Center | 45 | 2019-08-29 | 2026-09-25 |
| Single Agent and Combined Inhibition After Allogeneic Stem Cell Transplant Nivolumab, Ipilimumab - Acute Myeloid Leukemia and Myelodysplastic Syndrome | Phase 1 | Terminated | Hackensack Meridian Health | 8 | 2019-03-08 | 2024-11-18 |
| Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome Azacitidine, Cytarabine, Decitabine - Acute Myeloid Leukemia, Myelodysplastic Syndrome | Phase 2 / Phase 3 | Completed | National Cancer Institute (NCI) | 76 | 2018-02-02 | 2025-01-16 |
| Familial Investigations of Childhood Cancer Predisposition Acute Leukemia, Adenomatous Polyposis | Recruiting | St. Jude Children's Research Hospital | 1500 | 2017-04-06 | 2026-06-17 | |
| A Dose Escalation Phase I Study to Assess the Safety and Clinical Activity of Multiple Cancer Indications NKR-2 cells - Acute Myeloid Leukemia/Myelodysplastic Syndrome, Multiple Myeloma (MM) | Phase 1 / Phase 2 | Unknown | Celyad Oncology SA | 146 | 2016-12-01 | 2019-09-19 |
| Natural History Study of GATA2 Deficiency and Related Disorders GATA2 Deficiency | Recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 600 | 2013-08-26 | 2026-10-29 | |
| Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia Azacitidine, Laboratory Biomarker Analysis, Lenalidomide - Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1 | Phase 2 | Active not recruiting | National Cancer Institute (NCI) | 282 | 2012-03-01 | 2026-09-09 |
| Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC Cyclophosphamide (CTX, Cytoxan), Fludarabine(Fludara,Berlex Laboratories), Total Body Irradiation (TBI) - MDS, Immunodeficiency | Phase 2 | Completed | National Cancer Institute (NCI) | 19 | 2009-05-07 | 2020-03-12 |
Leukemia, Myeloid, Acute (8) · Myelodysplastic Syndromes (6) · Anemia, Refractory, with Excess of Blasts (3) · Leukemia, Myelomonocytic, Chronic (2) · Adenomatous Polyposis Coli (1) · Adrenocortical Carcinoma (1) · Anemia, Diamond-Blackfan (1) · Basal Cell Nevus Syndrome (1) · Blastic Plasmacytoid Dendritic Cell Neoplasm (1) · Burkitt Lymphoma (1) · Carney Complex (1) · Choroid Plexus Carcinoma (1) · Colorectal Neoplasms, Hereditary Nonpolyposis (1) · Dyskeratosis Congenita (1)
Bone Marrow Diseases (18) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (18) · Genetic Diseases, Inborn (18) · Hematologic Diseases (18) · Hemic and Lymphatic Diseases (18) · Neoplasms (13) · Neoplasms by Histologic Type (13) · Myelodysplastic Syndromes (12) · Leukemia (10) · Leukemia, Myeloid (9) · Immune System Diseases (5) · Anemia (4) · Anemia, Refractory (3) · Disease Attributes (3) · Immunoproliferative Disorders (3)
Azacitidine (5) · venetoclax (4) · Nivolumab (3) · Specimen Handling (3) · Cyclophosphamide (2) · Whole-Body Irradiation (2) · fludarabine phosphate (2) · Antilymphocyte Serum (1) · Biopsy (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cytarabine (1) · Decitabine (1) · Injections (1) · Ipilimumab (1)
National Cancer Institute (NCI) (4) · AB Science (1) · Apollo Therapeutics Ltd (1) · Celyad Oncology SA (1) · City of Hope Medical Center (1) · Federal Research Institute of Pediatric Hematology, Oncology and Immunology (1) · Fred Hutchinson Cancer Center (1) · Hackensack Meridian Health (1) · National Institute of Allergy and Infectious Diseases (NIAID) (1) · OHSU Knight Cancer Institute (1) · Qilu Pharmaceutical Co., Ltd. (1) · SCRI Development Innovations, LLC (1) · Servier (1) · Shanghai Bensen Pharmaceutical Co., Ltd. (1) · St. Jude Children's Research Hospital (1)
United States (13) · Australia (1) · Belgium (1) · Canada (1) · China (1) · France (1) · Greece (1) · Russia (1) · Spain (1) · United Kingdom (1)
Andrew Pecora, MD (1) · Curtis A Lachowiez (1) · Dennis D Hickstein, M.D. (1) · Evan C Chen (1) · James McCloskey, MD (1) · Jamie Koprivnikar, MD (1) · Kim E. Nichols, MD (1) · Laura C Michaelis (1) · Marie Bleakley (1) · Mikkael A Sekeres (1) · Nicholas Short, MD (1) · Norbert Vey, MD (1) · Paul Koller (1) · Sanjay Aggarwal, MD (1)
PMID 21508125, PMID 21816832, PMID 23344397, PMID 24077845, PMID 29133316, PMID 29618479, PMID 32556286, PMID 33049057, PMID 36764323, PMID 37595058, PMID 42013378
U10CA180888 (2), 2025 (1), P01CA018029 (1), P30CA015704 (1), P30CA033572 (1), U10CA032102 (1), UM1CA186709 (1)