Search clinical trials for: Inherited Bone Marrow Failure Syndrome

Total 75693 results

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
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
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
Sohag University
Assiut University; Kyoto University

Recruiting

Mutations and Phenotypes of Unclassifiable Inherited Bone Marrow Failure Syndromes

Inherited BMF Syndrome

Egypt
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
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
Assistance Publique - Hôpitaux de Paris

Recruiting

French National Registry of Bone Marrow Failures (RIME)

Bone Marrow Failure Syndrome

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
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
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
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
Children's Hospital of Philadelphia

Recruiting

Fludarabine Based RIC for Bone Marrow Failure Syndromes

Bone Marrow Failure Syndromes

United States
Masonic Cancer Center, University of Minnesota

Recruiting

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Sickle Cell Disease | Severe Congenital Neutropenia | Paroxysmal Nocturnal Hemoglobinuria | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Diamond Blackfan Anemia | Transfusion Dependent Alpha- or Beta- Thalassemia | Non-Malignant Hematologic Disorders

United States
Paul Szabolcs

Recruiting

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT (HSCT+RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias | Systemic Juvenile Idiopathic Arthritis (sJIA)

United States
Feinstein Institute for Medical Research
Diamond Blackfan Anemia Foundation, Inc.

Recruiting

Diamond Blackfan Anemia Registry (DBAR) (DBAR)

Anemia | Blood Disease

United States
National Cancer Institute (NCI)

Recruiting

Cancer in Inherited Bone Marrow Failure Syndromes

Fanconi Anemia | Dyskeratosis Congenita | Diamond Blackfan Anemia | Shwachman Diamond Syndrome | Inherited Bone Marrow Failure Syndrome, Aplastic Anemia

United States
Roswell Park Cancer Institute

Active, not recruiting

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

Acute Myeloid Leukemia | Polycythemia Vera | Myelofibrosis | Chronic Myelomonocytic Leukemia | Waldenstrom Macroglobulinemia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Secondary Acute Myeloid Leukemia | Sickle Cell Disease | Myelodysplastic Syndrome | Plasma Cell Myeloma | Chronic Granulomatous... and other conditions

United States
Bellicum Pharmaceuticals

Terminated

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

Hemoglobinopathies | Hemophagocytic Lymphohistiocytosis | Metabolic Disorders | Primary Immune Deficiency Disorders | Inherited Bone Marrow Failure Syndrome

United States
University of Florida
Live Like Bella Pediatric Cancer Research

Completed

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

Common Variable Immunodeficiency | Severe Combined Immunodeficiency | Sickle Cell Disease | Hurler Syndrome | Chronic Granulomatous Disease | Thalassemia | Wiskott-Aldrich Syndrome | Adrenoleukodystrophy | X-linked Lymphoproliferative Disease | Diamond Blackfan Anemia | Bone Marrow Failure Syndrome | Hemophagocytic... and other conditions

United States
Northwell Health

Terminated

Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia (ACE-011-DBA)

Diamond Blackfan Anemia

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

Completed

Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders

Non-Neoplastic Hematologic and Lymphocytic Disorder

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

Terminated

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders

Genetic Disorder | Severe Aplastic Anemia | Immunodeficiency Syndrome

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

Completed

Alemtuzumab, Fludarabine Phosphate, and Total-Body Irradiation Followed by a Donor Stem Cell Transplant in Treating Patients With Immunodeficiency or Other Nonmalignant Inherited Disorders

Immunodeficiency Syndrome | Non-Cancer Diagnosis

United States
Emory University

Completed

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Sickle Cell Disease | Fanconi Anemia | Hurler Syndrome | Chronic Granulomatous Disease | Severe Congenital Neutropenia | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Thalassemia Major | Leukocyte... and other conditions

United States
Roswell Park Cancer Institute

Completed

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Primary Myelofibrosis | Polycythemia Vera | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditions

United States
Emory University
Children's Healthcare of Atlanta

Terminated

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

Sickle Cell Disease | Fanconi Anemia | Thalassemia | Severe Congenital Neutropenia | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Leukocyte Adhesion Deficiency | Dyskeratosis-congenita | Chediak-Higashi Syndrome | Chronic-granulomatous Disease | Schwachman-Diamond...

United States
Children's Hospital Los Angeles
Lucile Packard Children's Hospital

Terminated

Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells

Metabolic Diseases | Stem Cell Transplantation | Chronic Granulomatous Disease | Bone Marrow Transplantation | Thalassemia | Wiskott-Aldrich Syndrome | Genetic Diseases | Peripheral Blood Stem Cell Transplantation | Pediatrics | Diamond-Blackfan Anemia | Allogeneic Transplantation | Combined Immune Deficiency | X-linked Lymphoproliferative Disease
Novartis

Completed

Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload

Myelodysplastic Syndromes | Hemoglobinopathies | Diamond-Blackfan Anemia | Transfusional Iron Overload | Other Inherited or Acquired Anaemia | MPD Syndrome | Other Rare Anaemias

Australia
National Center for Research Resources (NCRR)
James Whitcomb Riley Hospital for Children

Completed

Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

Thrombocytopenia | Dyskeratosis Congenita | Fanconi's Anemia | Shwachman Syndrome
Masonic Cancer Center, University of Minnesota

Not yet recruiting

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Acquired Pure Red Cell Aplasia | Paroxysmal Nocturnal Hemoglobinuria | Severe Aplastic Anemia | Acquired Amegakaryocytic Thrombocytopenia
Sidney Kimmel Comprehensive Cancer Center at Johns...
Janssen Research & Development, LLC

Not yet recruiting

Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies

Lymphoma | Multiple Myeloma | Hodgkin Lymphoma | Myelofibrosis | Non Hodgkin Lymphoma | Chronic Myeloid Leukemia | Hematologic Malignancy | Aplastic Anemia | Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukemia (AML) | Bone Marrow Transplant Rejection | Acute Lymphoblastic Leukemia (ALL), Adult
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
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
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
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
The First Affiliated Hospital of Soochow University

Recruiting

The Use of Eltrombopag Post HSCT in BMFS

Stem Cell Transplant Complications

China
Hugh Taylor

Recruiting

Bone Marrow Derived Stem Cells Mobilization for Treatment of Abnormal Endometrium

Asherman Syndrome | Recurrent Implantation Failure | Atrophic Endometrium

United States
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
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

Search clinical trials for: Inherited Bone Marrow Failure Syndrome

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