GATA2 deficiency with susceptibility to MDS/AML
GATA2 deficiency with susceptibility to MDS/AML
Explore linked clinical trials. Standard reference: MONDO:00429829 results ยท Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GATA2 deficiency with susceptibility to MDS/AML, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, bone marrow disorder, hematologic disorder, hereditary disease, leukemia, myelodysplastic syndrome, myeloid leukemia, neoplasm | Recruiting | Phase 1 | 2026-10-01 | NCT06484062 | |
| Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant | B-cell neoplasm, Burkitt Leukemia, Burkitt lymphoma, CD4+/CD56+ hematodermic neoplasm, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Virus Infections, Epstein-Barr virus infection, GATA2 deficiency with susceptibility to MDS/AML, Hemic and Lymphatic Diseases, Herpesviridae infectious disease, Infections, Mast Cell Activation Disorders, Mastocytosis, Systemic, Myeloproliferative Disorders, Neoplasms by Histologic Type, Neoplasms by Site, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Skin and Connective Tissue Diseases, Tumor Virus Infections, acute biphenotypic leukemia, acute leukemia, acute leukemia of ambiguous lineage, acute myeloid leukemia, acute undifferentiated leukemia, bone marrow disorder, hematologic disorder, hematopoietic and lymphoid system neoplasm, hereditary disease, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, malignant histiocytosis, mast cell leukemia, mastocytosis, mixed phenotype acute leukemia, myelodysplastic syndrome, myelodysplastic syndrome with excess blasts-1, myeloid leukemia, myeloproliferative neoplasm, neoplasm, skin disorder, skin neoplasm, viral infectious disease | Active, not recruiting | Phase 2 | 2026-09-25 | NCT03779854 | |
| Natural History Study of GATA2 Deficiency and Related Disorders | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Virus Infections, GATA2 deficiency with susceptibility to MDS/AML, Hemic and Lymphatic Diseases, Herpesviridae infectious disease, Infections, Neoplasms by Histologic Type, Skin Diseases, Viral, Skin and Connective Tissue Diseases, bone marrow disorder, hematologic disorder, hereditary disease, herpes simplex infectious disease, leukemia, myelodysplastic syndrome, neoplasm, skin disorder, skin disorder caused by infection, viral infectious disease | Recruiting | Not stated | 2026-09-22 | NCT01905826 | |
| Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia | Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, GATA2 deficiency with susceptibility to MDS/AML, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, anemia, bone marrow disorder, chronic myelomonocytic leukemia, hematologic disorder, hereditary disease, leukemia, myelodysplastic syndrome, myelodysplastic syndrome with excess blasts, myelodysplastic syndrome with excess blasts-1, myelodysplastic syndrome with single lineage dysplasia, myelodysplastic/myeloproliferative disease, myeloid leukemia, neoplasm | Active, not recruiting | Phase 2 | 2026-09-09 | NCT01522976 | |
| A Study to Evaluate S241656 Alone or in Combination in Participants With Selected Myeloid Malignancies | Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, GATA2 deficiency with susceptibility to MDS/AML, Hemic and Lymphatic Diseases, Myelodysplastic Syndrome Acute Myeloid Leukemia, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, acute myeloid leukemia, bone marrow disorder, chronic myelomonocytic leukemia, hematologic disorder, hereditary disease, leukemia, myelodysplastic syndrome, myelodysplastic/myeloproliferative disease, myeloid leukemia, neoplasm | Not yet recruiting | Phase 1 / Phase 2 | 2026-07-22 | NCT07719348 | |
| Iadademstat in Combination With Azacitidine and Venetoclax in Treating Newly Diagnosed Acute Myeloid Leukemia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GATA2 deficiency with susceptibility to MDS/AML, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, bone marrow disorder, hematologic disorder, hereditary disease, leukemia, myelodysplastic syndrome, myeloid leukemia, neoplasm | Recruiting | Phase 1 | 2026-06-30 | NCT06357182 | |
| Familial Investigations of Childhood Cancer Predisposition | Abnormalities, Multiple, Adenomatous Polyps, Adnexal Diseases, Bone Diseases, Developmental, Bone Marrow Failure Disorders, Cardiovascular Abnormalities, Carney complex, Ciliopathies, Colorectal Neoplasms, Hereditary Nonpolyposis, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cowden disease, Craniofacial Abnormalities, Cronkhite-Canada syndrome, Cysts, DICER1-related tumor predisposition, DNA repair disease, Diamond-Blackfan anemia, Digestive System Diseases, Digestive System Neoplasms, Disease Attributes, Ear Diseases, Eye Diseases, Hereditary, Fanconi anemia, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, GATA2 deficiency with susceptibility to MDS/AML, Genital Diseases, Hemic and Lymphatic Diseases, Hodgkins lymphoma, Hyperpigmentation, Infant, Newborn, Diseases, Jaw Cysts, Jaw Diseases, Li-Fraumeni syndrome, Lynch syndrome, Malformations of Cortical Development, Group I, Melanosis, Musculoskeletal Abnormalities, Myosarcoma, NF2-related schwannomatosis, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Multiple Primary, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Noonan syndrome, Nutritional and Metabolic Diseases, Otorhinolaryngologic Neoplasms, PTEN hamartoma tumor syndrome, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peutz-Jeghers syndrome, Rothmund-Thomson syndrome, Skin Abnormalities, Skin and Connective Tissue Diseases, Stomatognathic Diseases, Thoracic Neoplasms, Urogenital Diseases, Urogenital Neoplasms, X-linked disease, acoustic neuroma, acute leukemia, acute myeloid leukemia, adenocarcinoma, adenoma, adrenal cortex disorder, adrenal cortex neoplasm, adrenal gland cancer, adrenal gland disorder, anemia, angiomatosis, aplastic anemia, basal cell carcinoma, basal cell neoplasm, bone disorder, bone marrow disorder, breast disorder, breast neoplasm, carcinoma, cardiovascular disorder, central nervous system malformation, cerebral cortical dysplasia, choroid plexus carcinoma, classic familial adenomatous polyposis, colonic disorder, colorectal neoplasm, congenital heart disease, connective tissue disorder, connective tissue neoplasm, cranial nerve neoplasm, cranial nerve neuropathy, deafness-lymphedema-leukemia syndrome, digestive system disorder, digestive system neoplasm, disease susceptibility, dyskeratosis congenita, endocrine system disorder, epithelial neoplasm, eye disorder, familial rhabdoid tumor, female reproductive system disorder, female reproductive system neoplasm, gastrointestinal stromal tumor, gonadal disorder, hamartoma, heart cancer, heart disorder, hematologic disorder, hereditary Wilms tumor, hereditary breast ovarian cancer syndrome, hereditary disease, hereditary neoplastic syndrome, hereditary pheochromocytoma-paraganglioma, hereditary skin disorder, immune system disorder, immunoproliferative disorder, inherited aplastic anemia, inherited disease susceptibility, inherited neurodegenerative disorder, intestinal disorder, juvenile polyposis syndrome, lentigo, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, malignant endocrine neoplasm, malignant pancreatic neoplasm, melanocytic neoplasm, melanoma, metabolic disease, mismatch repair cancer syndrome, mismatch repair cancer syndrome 1, multiple endocrine neoplasia, multiple endocrine neoplasia type 1, multiple endocrine neoplasia type 2A, musculoskeletal system disorder, myelodysplastic syndrome, myomatous neoplasm, myxoma, neoplasm, nerve sheath neoplasm, nervous system disorder, neuroblastoma, neurocutaneous syndrome, neurodegenerative disease, neuroendocrine neoplasm, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuroma, neuromuscular disease, nevoid basal cell carcinoma syndrome, non-Hodgkin lymphoma, ocular cancer, odontogenic cyst, otorhinolaryngologic disease, ovarian cancer, ovarian disorder, pancreas disorder, pancreatic neoplasm, paraganglioma, peripheral nervous system disorder, pheochromocytoma, pheochromocytoma-paraganglioma, primitive neuroectodermal tumor, pure red-cell aplasia, retinal cancer, retinal disorder, retinoblastoma, retrocochlear disease, rhabdomyosarcoma, sarcoma, schwannoma, skin disorder, skin neoplasm, skin pigmentation disorder, solitary bone cyst, tuberous sclerosis, vascular disorder, vestibulocochlear nerve disorder, von Hippel-Lindau disease | Recruiting | Not stated | 2026-06-17 | NCT03050268 | |
| A Study to Investigate APL-4098 Alone and in Combination in Adults With AML or MDS | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GATA2 deficiency with susceptibility to MDS/AML, Hemic and Lymphatic Diseases, Myelodysplastic Syndrome Acute Myeloid Leukemia, Neoplasms by Histologic Type, acute myeloid leukemia, anemia, bone marrow disorder, hematologic disorder, hereditary disease, leukemia, myelodysplastic syndrome, myelodysplastic syndrome with excess blasts, myelodysplastic syndrome with single lineage dysplasia, myeloid leukemia, neoplasm | Recruiting | Phase 1 | 2026-06-01 | NCT06372717 | |
| AB8939 in Patients With Relapsed/Refractory Acute Myeloid Leukemia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GATA2 deficiency with susceptibility to MDS/AML, Hemic and Lymphatic Diseases, Myelodysplastic Syndrome Acute Myeloid Leukemia, Neoplasms by Histologic Type, acute myeloid leukemia, bone marrow disorder, hematologic disorder, hereditary disease, leukemia, myelodysplastic syndrome, myeloid leukemia, neoplasm | Recruiting | Phase 1 / Phase 2 | 2025-10-02 | NCT05211570 |