Clinical Trials on Congenital Bone Marrow Failure Syndromes

Total 12434 results

Sohag University
Assiut University; Kyoto University

Recruiting

Mutations and Phenotypes of Unclassifiable Inherited Bone Marrow Failure Syndromes

Inherited BMF Syndrome

Egypt
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

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
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
University of California, San Francisco

Terminated

Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation

Myeloid Malignancy | Metabolic Disease | Bone Marrow Failure Syndrome | Transfusion-dependent Red Blood Cell (RBC) Defect | Congenital Immunodeficiency | Severe Immune Dysregulation

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
Boston Children's Hospital
Massachusetts General Hospital; Mayo Clinic; Karolinska University Hospital; Baylor... and other collaborators

Active, not recruiting

Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

Aplastic Anemia | Dyskeratosis Congenita | Hoyeraal Hreidarsson Syndrome | Revesz Syndrome

United States, Norway, Sweden
Children's Hospital of Philadelphia

Available

Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC

Leukemia | Immunodeficiencies | Bone Marrow Failure Syndrome

United States
St. Jude Children's Research Hospital

Active, not recruiting

Haploidentical HCT for Severe Aplastic Anemia

Aplastic Anemia | Bone Marrow Failure Syndrome

United States
University of Calgary

Unknown

The Impact of Red Cell Age on Product Utilization in the Chronically Transfused Outpatient Population (ABC-TOP)

Bone Marrow Failure Syndrome
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
Rabin Medical Center

Unknown

Eltrombopag for Enhancing Platelet Engraftment in Adult Patients Undergoing Cord Blood Transplantation

Hematological Malignancy | Bone Marrow Failure Syndrome

Israel
Children's Hospital of Philadelphia

Completed

Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases (MiniClini)

Bone Marrow Failure Syndromes | Immunodeficiencies | Immune Dysregulation Syndromes

United States
University of British Columbia

Completed

Umbilical Cord Blood Transplantation As Treatment Of Adult Patients With Hematologic Disorders

Hematological Malignancy | Bone Marrow Failure Syndrome

Canada
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
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
National Cancer Institute (NCI)

Completed

Needs Assessment for Individuals and Families Affected by Dyskeratosis Congenita (DC) and Related Telomere Biology Disorders (TBD)

Dyskeratosis Congenita | Telomere Biology Disorders

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
Cellenkos, Inc.

Active, not recruiting

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)

Bone Marrow Disease

United States
Masonic Cancer Center, University of Minnesota

Recruiting

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

Aplastic Anemia | Dyskeratosis Congenita

United States
Masonic Cancer Center, University of Minnesota

Completed

Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA

Aplastic Anemia | Dyskeratosis Congenita

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

Recruiting

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells

MDS (Myelodysplastic Syndrome) | Severe Aplastic Anemia

United States
Talaris Therapeutics Inc.
Duke University

Withdrawn

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

Anemia, Sickle Cell | Thalassemia | Beta-Thalassemia | Bone Marrow Failure Syndromes | Diamond-Blackfan Anemia | Alpha-Thalassemia | Complex and Transfusion-dependent Hemoglobinopathies

United States
Children's Hospital of Philadelphia

Recruiting

Fludarabine Based RIC for Bone Marrow Failure Syndromes

Bone Marrow Failure Syndromes

United States
Medical College of Wisconsin
Children's Hospital of Philadelphia

Completed

Peripheral Blood Stem Cell Transplantation Using the CliniMACS Device

Malignant Diseases (ie, Leukemia, MDS, Lymphoma) | Non-malignant Diseases (ie, Bone Marrow Failure Syndromes)

United States
St. Jude Children's Research Hospital
Baylor College of Medicine; Children's Hospital of Philadelphia; Dana-Farber... and other collaborators

Recruiting

Investigation of the Genetics of Hematologic Diseases

Myelodysplastic Syndromes | Sickle Cell Disease | Fanconi Anemia | Severe Congenital Neutropenia | Bone Marrow Failure Syndromes | Blood Coagulation Disorder | Erythrocyte Disorder | Leukocyte Disorder | Hemostasis | Dyskeratosis Congenita | Diamond-Blackfan Anemia | Congenital Thrombocytopenia | Myeloproliferative...

United States
Northwell Health

Completed

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia (LeucineDBA)

Pure Red Cell Aplasia | Diamond Blackfan Anemia | Blackfan Diamond Syndrome | DBA | Congenital Hypoplastic Anemia

United States
University of California, San Francisco
National Cancer Institute (NCI)

Completed

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

Myelodysplastic Syndromes | Leukemia | Congenital Amegakaryocytic Thrombocytopenia | Severe Congenital Neutropenia | Diamond-blackfan Anemia

United States
Memorial Sloan Kettering Cancer Center
Boston Children's Hospital; Fred Hutchinson Cancer Center; Children's Hospital... and other collaborators

Completed

Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

Leukemia | Myelodysplastic Syndrome | Aplastic 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
Sidney Kimmel Comprehensive Cancer Center at Johns...

Completed

Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia

Bone Marrow Failure Syndromes | Severe Aplastic Anemia

United States
University Hospital Freiburg

Completed

Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

Myelodysplastic Syndromes | Fanconi Anemia | Dyskeratosis Congenita | Pearson Marrow-pancreas Syndrome | Shwachman-diamond Syndrome

Spain, Germany, Switzerland, Austria, Netherlands, Italy, Czech Republic, Belgium, Denmark, Ireland
Xiaofan Zhu

Available

Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their Patents

Fanconi Anemia | Autosomal or Sex Linked Recessive Genetic Disease | Bone Marrow Hematopoiesis Failure, Multiple Congenital Abnormalities, and Susceptibility to Neoplastic Diseases. | Hematopoiesis Maintainance.

China
Cooperative Study Group A for Hematology

Completed

Comparison of Cy-Atg Vs Cy-Flu-Atg for the Conditioning Therapy in Allo-HCT (CyFluCyATG)

Bone Marrow Failure Syndromes

Korea, Republic of
National Heart, Lung, and Blood Institute (NHLBI)

Active, not recruiting

Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

Anemia, Diamond-Blackfan

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
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia

Diamond Blackfan Anemia

United States
St. Jude Children's Research Hospital

Completed

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

Anemia, Aplastic | Diamond-Blackfan Anemia | Amegakaryocytic Thrombocytopenia | Kostmann Syndrome

United States
Children's Hospital Medical Center, Cincinnati
FDA Office of Orphan Products Development

Completed

Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

Fanconi Anemia

United States
Alexion

Completed

Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure Syndromes (EXPLORE)

Hemoglobinuria
Federal Scientific Clinical Centre of Pediatric...

Unknown

L-leucine in Diamond Blackfan Anemia Patients

Diamond Blackfan Anemia

Russian Federation
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
Handok Inc.

Unknown

Observational of PNH Type Cells in Korean Patients With Bone Marrow Failure Syndrome and Having Hemolytic PNH (OPENK)

PNH
Jason Robert Gotlib
Celgene Corporation

Terminated

Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia

Leukemia | Anemia | Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukemia (AML)

United States
Asan Medical Center

Completed

Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome

Myelodysplastic Syndromes | Leukemia | Nonmalignant Neoplasm | Paroxysmal Nocturnal Hemoglobinuria

Korea, Republic of
Emory University

Terminated

G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome (G-CSF PMRD)

Hematologic Malignancies

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

Recruiting

Diamond Blackfan Anemia Registry (DBAR) (DBAR)

Anemia | Blood Disease

United States
Peter MacCallum Cancer Centre, Australia

Recruiting

The Evaluating Multidisciplinary Bone Marrow Failure Care in Bone Marrow Failure and Related Disorders. (EMBRACE)

Aplastic Anemia | Bone Marrow Failure Syndrome

Australia
Boston Children's Hospital

Terminated

Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita

Fanconi Anemia | Dyskeratosis Congenita

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

Clinical Trials on Congenital Bone Marrow Failure Syndromes

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