Search clinical trials for: bone marrow failure syndromes

Total 55591 results

National Cancer Institute (NCI)

Not yet recruiting

Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms

Myelodysplastic Syndromes

United States
Peking University People's Hospital

Not yet recruiting

Sirolimus+Abatacept+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to Calcineurin Inhibitors

Myelodysplastic Syndromes | Acute Leukemia | Severe Aplastic Anemia
Hospital Universitari Vall d'Hebron Research Institute
Erasme University Hospital; Cyprus Institute of Neurology and Genetics; EuroBloodNet...

Recruiting

RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs) (RADeep)

Iron Metabolism Disorders | Sickle Cell Disease | Thalassemia | Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder | Anemia Due to Membrane Defect | CDA | Sideroblastic Anemia | Constitutional Aplastic Anemia | Hereditary Anemia

Spain
National Cancer Institute (NCI)

Recruiting

Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

Inherited Bone Marrow Failure Syndrome | Familial Platelet Disorder With Predisposition to Myeloid Malignancies

United States
UK Kidney Association

Recruiting

National Registry of Rare Kidney Diseases (RaDaR)

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom
Boston Children's Hospital
Children's Hospital Medical Center, Cincinnati

Recruiting

Shwachman Diamond Syndrome Registry and Study (SDSR)

Shwachman-Diamond Syndrome | Shwachman-Diamond Syndrome-Like

United States
University of Alabama at Birmingham

Not yet recruiting

CD34 Selection Using the Automated CliniMACS Prodigy

Myelodysplastic Syndromes | Primary Myelofibrosis | AML | Lymphoid Malignancies | CML | ALL

United States
Masonic Cancer Center, University of Minnesota
National Institutes of Health (NIH)

Recruiting

Role of Acetaldehyde in the Development of Oral Cancer

Fanconi Anemia | Oral Cavity Carcinoma | Alcohol-Related Carcinoma

United States
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Observational Follow-up Study of Haplo-identical Transplants in Fanconi Disease (HAPLO-FANCONI)

Fanconi Syndrome
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia

Steroid-refractory Diamond-Blackfan Anemia (DBA)

United States
Shandong Qilu Stem Cells Engineering Co., Ltd.
Second Affiliated Hospital of Zhengzhou University; The Second Affiliated Hospital... and other collaborators

Recruiting

Clinical Study of UCB Combined With UC-MSCs in the Treatment of Bone Marrow Failure Disorders

Bone Marrow Failure Disorders

China
Franziska Wachter
Harvard Clinical and Translational Science Center (Harvard Catalyst)

Recruiting

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 (MORE)

Acute Myeloid Leukemia | Myelodysplastic Syndromes | MDS | Aml | Myeloid Neoplasm | Myeloid Malignancies | Inherited Bone Marrow Failure Syndrome

United States
National Cancer Institute (NCI)

Recruiting

Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia

Fanconi Anemia | Inherited Bone Marrow Failure Syndrome

United States
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
Children's Hospital Medical Center, Cincinnati

Recruiting

Time-restricted Feeding to Reduce Inflammation in Fanconi Anemia

Fanconi Anemia

United States
Thomas Jefferson University
AbbVie

Recruiting

Evaluating Navitoclax After Failure of Standard Treatments of Azacitidine or Decitabine and Venetoclax in Patients With Aggressive Myelodysplastic Syndrome

Myelodysplastic Syndrome | Recurrent Myelodysplastic Syndrome | Refractory Myelodysplastic Syndrome

United States
Sohag University
Assiut University; Kyoto University

Recruiting

Mutations and Phenotypes of Unclassifiable Inherited Bone Marrow Failure Syndromes

Inherited BMF Syndrome

Egypt
Centre Leon Berard

Recruiting

Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation (EVAADE)

Myelodysplastic Syndromes | Severe Combined Immunodeficiency | Acute Leukemia | Haematological Malignancy | Hemoglobinopathy in Children | Bone Marrow Aplasia

France
Baylor College of Medicine
The Methodist Hospital Research Institute; Center for Cell and Gene Therapy...

Recruiting

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion (HAPLOTAB)

Myelodysplastic Syndromes | Primary Immunodeficiency Diseases | Hemoglobinopathies | Chronic Myeloid Leukemia | Cytopenia | Severe Aplastic Anemia | Bone Marrow Failure Syndrome | Acute Myeloid Leukemia in Remission | Hemophagocytic Lymphohistiocytoses | Acute Lymphoblastic Leukemia in Remission | Severe...

United States
Peter MacCallum Cancer Centre, Australia
National Health and Medical Research Council, Australia; University of Melbourne

Recruiting

Diagnosis and Phenotype Characterisation Using Genomics in Patients With Inherited Bone Marrow Failure (IBMDx Study) (IBMDx)

Hematologic Diseases | Inherited Platelet Disorder | Inherited BMF Syndrome

Australia
Children's Hospital of Philadelphia
National Cancer Institute (NCI); University of Pennsylvania; City of Hope Medical... and other collaborators

Recruiting

Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors (IAMFIT)

Myelodysplastic Syndromes | Aplastic Anemia | Lymphoblastic Leukemia | Cancer Survivors

United States
Thomas Jefferson University

Recruiting

Reduced-Intensity Conditioning for the Prevention of Treatment-Related Mortality in Patients Who Undergo a Hematopoietic Stem Cell Transplant

Hematopoietic and Lymphoid Cell Neoplasm | Acute Myeloid Leukemia | Myelodysplastic Syndromes | Myeloid Leukemia | Polycythemia Vera | Essential Thrombocythemia | Hodgkin Lymphoma | Myelofibrosis | Chronic Myelomonocytic Leukemia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Non-Hodgkin Lymphoma and other conditions

United States
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

Severe Aplastic Anemia | Telomere Biology Disorders | Inherited Bone Marrow Failure Syndromes

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaborators

Recruiting

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

Congenital Amegakaryocytic Thrombocytopenia | Paroxysmal Nocturnal Hemoglobinuria | Shwachman-Diamond Syndrome | Bone Marrow Failure Syndrome | Congenital Pure Red Cell Aplasia | Hereditary Sideroblastic Anemia | Hematologic Neoplasm With Germline GATA2 Mutation | Hematologic Neoplasm With Germline... and other conditions

United States
Porteus, Matthew, MD

Recruiting

Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab

Fanconi Anemia

United States
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Microcephaly, Fanconi Anemia and Praxial Disorders (MicroFancII)

Fanconi Anemia

France
Institute of Hematology & Blood Diseases Hospital

Recruiting

National Longitudinal Cohort of Hematological Diseases (NICHE)

Lymphoma | Acute Myeloid Leukemia | Leukemia | Hemophilia A | Hemophilia B | Hemophilia | Multiple Myeloma | Myelodysplastic Syndrome | Bone Marrow Transplantation | MDS | Aplastic Anemia | Bleeding Disorder | Myeloma, Multiple | Myelomas, Multiple | Leukemias, Acute Myeloid | Myeloid Leukemias, Acute | Hemophilia As | Hemophilia... and other conditions

China
The University of Queensland

Recruiting

The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary Fibrosis

Pulmonary Fibrosis | Dyskeratosis Congenita | Telomere Shortening | Telomere Disease

Australia
Paul Szabolcs

Recruiting

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC (PRO-RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias

United States
Foresee Pharmaceuticals Co., Ltd.

Recruiting

A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia

Fanconi Anemia

United States
Johns Hopkins All Children's Hospital

Recruiting

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

Severe Aplastic Anemia | Bone Marrow Failure Syndrome | Hemoglobinopathy (Disorder)

United States
Washington University School of Medicine
Shwachman Diamond Syndrome Foundation; Barnes-Jewish Hospital

Recruiting

Diabetes/ Endocrine Surveillance in SDS

Shwachman-Diamond Syndrome

United States
Sidney Kimmel Comprehensive Cancer Center at Johns...

Recruiting

Reduced Intensity BMT for Immune Dysregulatory and Bone Marrow Failure Syndromes Using Post-Transplant Cyclophosphamide

Primary Immune Deficiency Disorder | Bone Marrow Failure | Immune Deficiency Disease

United States
Mitchell Cairo

Recruiting

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Hodgkin Lymphoma | Sickle Cell Disease | Acute Leukemia | Beta-Thalassemia | Non-hodgkin Lymphoma | Severe Aplastic Anemia | Diamond Blackfan Anemia | Amegakaryocytic Thrombocytopenia | Kostmann

United States
National Taiwan University Hospital

Recruiting

Screening Gene Mutations in Myeloid Cancers by Next Generation Sequencing to Improve Treatment Results

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Aplastic Anemia | Myeloproliferative Neoplasm

Taiwan
Academic and Community Cancer Research United
National Cancer Institute (NCI)

Recruiting

Olaparib in Treating Patients With Metastatic Biliary Tract Cancer With Aberrant DNA Repair Gene Mutations

Bile Duct Adenocarcinoma | Fanconi Anemia Complementation Group Gene Mutation | Metastatic Bile Duct Carcinoma | PTEN Gene Deletion

United States
Case Comprehensive Cancer Center

Recruiting

CPX-351 Therapy for MDS After Hypomethylating Agent Failure

Myelodysplastic Syndrome (MDS)

United States
University College, London
Medical Research Council

Recruiting

Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation (ToTem)

Lymphoma | Myelodysplastic Syndromes | Leukemia | Myeloma | Graft Vs Host Disease | Severe Aplastic Anemia | Primary Immune Deficiency

United Kingdom
Paul Szabolcs

Recruiting

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Sickle Cell Anemia | Diamond-blackfan Anemia | Beta-thalassemia Major

United States
M.D. Anderson Cancer Center

Recruiting

Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant

Follicular Lymphoma | Acute Myeloid Leukemia | Hodgkin Lymphoma | Acute Lymphoblastic Leukemia | Mantle Cell Lymphoma | Chronic Lymphocytic Leukemia | Non-Hodgkin Lymphoma | Myelodysplastic Syndrome | Plasma Cell Myeloma | Aplastic Anemia | Myeloproliferative Neoplasm | Bone Marrow Failure | Chronic Myelogenous...

United States
Masonic Cancer Center, University of Minnesota

Recruiting

T Cell Receptor α/β TCD HCT in Patients With Fanconi Anemia

Myelodysplastic Syndromes | Fanconi Anemia | Severe Aplastic Anemia

United States
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Paroxysmal Nocturnal Hemoglobinuria (PNH) | Severe Aplastic Anemia (SAA) | Hypo-Plastic Myelodysplastic Syndrome (MDS)

United States
Masonic Cancer Center, University of Minnesota

Recruiting

Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders

Lymphoma | Follicular Lymphoma | Acute Myeloid Leukemia | Multiple Myeloma | Myelofibrosis | Juvenile Myelomonocytic Leukemia | Burkitt Lymphoma | Acute Lymphoblastic Leukemia | Lymphoblastic Lymphoma | Chronic Lymphocytic Leukemia | Lymphoplasmacytic Lymphoma | Acute Leukemia | Mantle-Cell Lymphoma | Chronic Myelogenous... and other conditions

United States
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Eltrombopag for People With Fanconi Anemia

Fanconi Anemia

United States
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome

Myelodysplastic Syndrome (MDS) | Severe Aplastic Anemia | Hypo-Plastic MDS

United States
Washington University School of Medicine

Recruiting

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

Hemoglobinopathies | Bone Marrow Failure Syndromes | Metabolic Disorders | Non-malignant Disorders | Severe Sickle Cell Disease | Immunologic Disorders

United States
St. Jude Children's Research Hospital

Recruiting

Familial Investigations of Childhood Cancer Predisposition (SJFAMILY)

Pancreatic Cancer | Hodgkin Lymphoma | Lynch Syndrome | Tuberous Sclerosis | Fanconi Anemia | AML | Non Hodgkin Lymphoma | Familial Adenomatous Polyposis | Acute Leukemia | Nevoid Basal Cell Carcinoma Syndrome | Neurofibromatosis Type 1 | Neuroblastoma | Retinoblastoma | MDS | Rhabdomyosarcoma | Von Hippel-Lindau Disease | Adrenocortical Carcinoma and other conditions

United States
Children's Hospital of Philadelphia

Recruiting

Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF

Paroxysmal Nocturnal Hemoglobinuria | Acquired Aplastic Anemia | Inherited Bone Marrow Failure Syndromes

United States
University of Rochester

Recruiting

Umbilical Cord Blood Transplantation From Unrelated Donors

Lymphoma | Myelodysplastic Syndromes | Multiple Myeloma | Solid Tumor | Acute Leukemia | Aplastic Anemia | Chronic Leukemia | Immune Deficiency Disorder | Congenital Hematological Disorder | Metabolism Disorder

United States
Mayo Clinic

Recruiting

Pre-myeloid Cancer and Bone Marrow Failure Clinic Study

Hematologic Neoplasms | Myeloid Malignancy | Cytopenia | Bone Marrow Failure Syndrome | Clonal Cytopenia of Undetermined Significance | Clonal Hematopoiesis of Indeterminate Potential | Low Risk Myelodysplastic Syndrome | Inherited Bone Marrow Failure Syndrome | Clonal Expansion | Hereditary Neoplastic... and other conditions

United States

Search clinical trials for: bone marrow failure syndromes

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Singapore

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations