Congenital Bone Marrow Failure Syndromes
Congenital Bone Marrow Failure Syndromes
Explore linked clinical trials. Standard reference: MESH:M00064919541 results · Page 1 of 2
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Clinical Genetics Branch Eligibility Screening Survey | Abnormalities, Multiple, Bone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Costello syndrome, Craniofacial Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Legius syndrome, Li-Fraumeni syndrome, Musculoskeletal Abnormalities, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, Thoracic Neoplasms, anemia, aplastic anemia, bone marrow disorder, cardiofaciocutaneous syndrome, chordoma, hematologic disorder, hereditary disease, hereditary neoplastic syndrome, inherited aplastic anemia, lung neoplasm, melanocytic neoplasm, melanoma, metabolic disease, mixed neoplasm, musculoskeletal system disorder, neoplasm, neuroendocrine neoplasm, primitive neuroectodermal tumor, pulmonary blastoma, respiratory tract neoplasm, skin disorder, skin neoplasm, susceptibility to familial cutaneous melanoma | Not yet recruiting | Not stated | 2026-10-09 | NCT07005297 | |
| Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, bone marrow disorder, hematologic disorder | Recruiting | Phase 1 | 2026-10-08 | NCT06090669 | |
| Cancer in Inherited Bone Marrow Failure Syndromes | Bone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Diamond-Blackfan anemia, Digestive System Diseases, Fanconi anemia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Shwachman-Diamond syndrome, Skin Abnormalities, Skin and Connective Tissue Diseases, X-linked disease, anemia, aplastic anemia, bone marrow disorder, dyskeratosis congenita, exocrine pancreatic insufficiency, hematologic disorder, hereditary disease, hereditary skin disorder, inherited aplastic anemia, lipomatosis, metabolic disease, pancreas disorder, pure red-cell aplasia, skin disorder | Recruiting | Not stated | 2026-10-08 | NCT00027274 | |
| Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease, neoplasm | Active, not recruiting | Not stated | 2026-10-02 | NCT06744283 | |
| Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Nutritional and Metabolic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease | Recruiting | Not stated | 2026-10-01 | NCT05687149 | |
| Natural History of Acquired and Inherited Bone Marrow Failure Syndromes | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder | Recruiting | Not stated | 2026-09-30 | NCT05012111 | |
| Eltrombopag for Fanconi Anemia | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Inflammation, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease, pancytopenia | Active, not recruiting | Phase 2 | 2026-09-16 | NCT03206086 | |
| Widefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at Risk for Developing Oral Cancer | Bone Marrow Failure Disorders, Chemically-Induced Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Erythroplasia, Fanconi anemia, Head and Neck Neoplasms, Hemic and Lymphatic Diseases, Lichenoid Eruptions, Mouth Neoplasms, Neoplasms by Site, Nutritional and Metabolic Diseases, Skin Diseases, Papulosquamous, Skin and Connective Tissue Diseases, Stomatognathic Diseases, anemia, aplastic anemia, bone marrow disorder, erythroplakia, hematologic disorder, hereditary disease, hereditary mucosal leukokeratosis, hereditary skin disorder, inherited aplastic anemia, lichen planus, metabolic disease, mouth disorder, neoplasm, nicotine dependence, precancerous condition, psychiatric disorder, skin disorder, substance-related disorder | Active, not recruiting | Not applicable | 2026-09-14 | NCT00542373 | |
| Quercetin Dyskeratosis Congenita (DC)/Telomere Biology Disorders (TBD) | Bone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, X-linked disease, bone marrow disorder, dyskeratosis congenita, hematologic disorder, hereditary disease, hereditary skin disorder, skin disorder | Recruiting | Phase 1 | 2026-09-10 | NCT07628972 | |
| A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, bone marrow disorder, hematologic disorder, leukemia, myelodysplastic syndrome, myeloid leukemia, myeloid neoplasm, neoplasm | Active, not recruiting | Phase 2 | 2026-08-20 | NCT05796570 | |
| T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diamond-Blackfan anemia, Hemic and Lymphatic Diseases, anemia, aplastic anemia, beta thalassemia, beta-thalassemia major, bone marrow disorder, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited aplastic anemia, inherited hemoglobinopathy, pure red-cell aplasia, sickle cell disease, thalassemia | Recruiting | Phase 1 / Phase 2 | 2026-08-13 | NCT03653338 | |
| A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections | Abnormalities, Multiple, Barth syndrome, Bone Marrow Failure Disorders, Cardiovascular Abnormalities, Cohen syndrome, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Digestive System Diseases, Hemic and Lymphatic Diseases, Kostmann syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Shwachman-Diamond syndrome, X-linked disease, agranulocytosis, autoimmune disease, bone marrow disorder, cardiovascular disorder, congenital heart disease, exocrine pancreatic insufficiency, heart disorder, hematologic disorder, hereditary disease, immune system disorder, inborn errors of metabolism, leukocyte disorder, leukopenia, lipomatosis, metabolic disease, neutropenia, pancreas disorder | Recruiting | Phase 3 | 2026-08-06 | NCT06056297 | |
| Nucleoside Therapy in Patients With Telomere Biology Disorders | Bone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fibrosis, Hemic and Lymphatic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Revesz syndrome, Skin Abnormalities, Skin and Connective Tissue Diseases, X-linked disease, bone marrow disorder, dyskeratosis congenita, hematologic disorder, hereditary disease, hereditary skin disorder, interstitial lung disease, lung disorder, pulmonary fibrosis, respiratory system disorder, skin disorder | Recruiting | Phase 1 | 2026-08-04 | NCT06817590 | |
| Role of Acetaldehyde in the Development of Oral Cancer | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Head and Neck Neoplasms, Hemic and Lymphatic Diseases, Mouth Neoplasms, Neoplasms by Site, Nutritional and Metabolic Diseases, Stomatognathic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease, mouth disorder, neoplasm, oral cavity carcinoma | Recruiting | Not applicable | 2026-07-22 | NCT05973656 | |
| Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diamond-Blackfan anemia, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, pure red-cell aplasia | Recruiting | Phase 1 | 2026-07-21 | NCT07476183 | |
| MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease | Recruiting | Not stated | 2026-07-02 | NCT07649031 | |
| 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 | |
| Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, Infant, Newborn, Diseases, acquired aplastic anemia, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuria | Active, not recruiting | Not applicable | 2026-06-17 | NCT03047746 | |
| Prenatal Transplantation for Fetuses With Fanconi Anemia | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Nutritional and Metabolic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease | Not yet recruiting | Phase 1 / Phase 2 | 2026-06-16 | NCT07408583 | |
| Investigation of the Genetics of Hematologic Diseases | Bone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Diamond-Blackfan anemia, Fanconi anemia, Hemic and Lymphatic Diseases, Kostmann syndrome, Nutritional and Metabolic Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, X-linked disease, anemia, aplastic anemia, blood coagulation disease, bone marrow disorder, dyskeratosis congenita, erythrocyte disorder, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, hereditary skin disorder, inherited aplastic anemia, inherited hemoglobinopathy, leukocyte disorder, metabolic disease, myelodysplastic syndrome, pure red-cell aplasia, sickle cell disease, skin disorder | Recruiting | Not stated | 2026-06-09 | NCT02720679 | |
| Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC) | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Head and Neck Neoplasms, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Nutritional and Metabolic Diseases, anemia, aplastic anemia, bone marrow disorder, carcinoma, epithelial neoplasm, head and neck squamous cell carcinoma, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease, neoplasm, squamous cell carcinoma | Recruiting | Phase 1 / Phase 2 | 2026-05-27 | NCT06648096 | |
| Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS) | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diamond-Blackfan anemia, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, pure red-cell aplasia | Recruiting | Not stated | 2026-04-27 | NCT07186179 | |
| Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT | Bone Marrow Failure, Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Neoplasms by Histologic Type, Nutritional and Metabolic Diseases, acquired aplastic anemia, anemia, bone marrow disorder, hematologic disorder, hemophagocytic syndrome, hereditary disease, immune system disorder, immunodeficiency disease, immunoproliferative disorder, inborn error of immunity, inborn errors of metabolism, inherited hemoglobinopathy, leukemia, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, metabolic disease, myelodysplastic syndrome, myelodysplastic syndrome with excess blasts, myelodysplastic syndrome with single lineage dysplasia, neoplasm | Recruiting | Phase 1 / Phase 2 | 2026-04-15 | NCT06839456 | |
| Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102 | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease | Enrolling by invitation | Not stated | 2026-04-14 | NCT07527975 | |
| Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Fanconi anemia complementation group A, Fanconi renotubular syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, kidney disorder, metabolic disease, renal tubular transport disease, urinary system disorder | Active, not recruiting | Not stated | 2026-04-13 | NCT04437771 |