Search clinical trials for: Inherited Bone Marrow Failure Syndromes

Total 57412 results

Jennifer Woyach
Janssen Scientific Affairs, LLC

Completed

Ibrutinib for the Treatment of COVID-19 in Patients Requiring Hospitalization

Hematopoietic and Lymphoid Cell Neoplasm | Malignant Solid Neoplasm | Monoclonal Gammopathy of Undetermined Significance | Myelodysplastic Syndrome | Aplastic Anemia | Symptomatic COVID-19 Infection Laboratory-Confirmed | Monoclonal B-Cell Lymphocytosis

United States
Center for International Blood and Marrow Transplant...
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaborators

Active, not recruiting

Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation (BMT CTN 1702) (BMT CTN 1702)

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Hodgkin Lymphoma | Acute Lymphoblastic Leukemia | Sickle Cell Disease | Non-hodgkin Lymphoma | Acquired Aplastic Anemia

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
Paul Szabolcs

Enrolling by invitation

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases (BOLT+BMT)

Chronic Granulomatous Disease (CGD) | Wiskott-Aldrich Syndrome | Severe Chronic Neutropenia | Severe Combined Immunodeficiency (SCID) | Immunodeficiency With Predominant T-cell Defect, Unspecified | Hyper IgE Syndromes | Hyper IgM Deficiencies | Mendelian Susceptibility to Mycobacterial Disease | Common...

United States
M.D. Anderson Cancer Center

Completed

DS-1594b With or Without Azacitidine, Venetoclax, or Mini-HCVD for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia or Acute Lymphoblastic Leukemia

Hematopoietic and Lymphoid Cell Neoplasm | Recurrent Acute Myeloid Leukemia | Recurrent Chronic Myelomonocytic Leukemia | Recurrent Myelodysplastic Syndrome | Refractory Acute Myeloid Leukemia | Refractory Chronic Myelomonocytic Leukemia | Refractory Myelodysplastic Syndrome | Recurrent Acute Lymphoblastic... and other conditions

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

Active, not recruiting

Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

Anemia, Diamond-Blackfan

United States
medac GmbH
Syneos Health; Celerion; Venn Life Sciences

Completed

Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases

Bone Marrow Failure Syndromes | Inborn Errors of Metabolism | Primary Immunodeficiencies | Haemoglobinopathies

Poland, Czechia, Germany, Italy
Children's Hospital Medical Center, Cincinnati

Active, not recruiting

Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

Fanconi Anemia | Squamous Cell Carcinoma

United States
Bellicum Pharmaceuticals

Terminated

Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant

Lymphoma, Non-Hodgkin | Acute Lymphoblastic Leukemia | Primary Immunodeficiency | Anemia, Sickle Cell | Hemoglobinopathies | Fanconi Anemia | Anemia, Aplastic | Myelodysplastic Syndrome | Thalassemia | Cytopenia | Diamond Blackfan Anemia | Osteopetrosis | Leukemia, Acute Myeloid (AML), Child

United Kingdom, Italy
Bellicum Pharmaceuticals

Terminated

Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study

Lymphoma, Non-Hodgkin | Myelodysplastic Syndromes | Acute Lymphoblastic Leukemia | Primary Immunodeficiency | Anemia, Sickle Cell | Hemoglobinopathies | Fanconi Anemia | Anemia, Aplastic | Thalassemia | Cytopenia | Diamond Blackfan Anemia | Leukemia, Acute Myeloid (AML), Child

Italy
University of California, San Francisco

Completed

Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

Sickle Cell Disease | Hemoglobinopathies | Fanconi Anemia | Thalassemia | Hematologic Malignancies | Immune Deficiency | Nonmalignant Diseases | Genetic Inborn Errors of Metabolism

United States
Julian Sevilla

Completed

Eltrombopag for Treatment of Fanconi Anemia

Fanconi Anemia

Spain
University of Sao Paulo
Conselho Nacional de Desenvolvimento Científico e Tecnológico

Completed

Nandrolone Decanoate in the Treatment of Telomeropathies

Idiopathic Pulmonary Fibrosis | Aplastic Anemia | Bone Marrow Failure Syndromes | Telomere Shortening

Brazil
St. Jude Children's Research Hospital

Active, not recruiting

Haploidentical HCT for Severe Aplastic Anemia

Aplastic Anemia | Bone Marrow Failure Syndrome

United States
Ohio State University Comprehensive Cancer Center

Active, not recruiting

Geriatric Optimization Plan to Improve Survival in Older Adult Allogeneic Hematopoietic Cell Transplant Candidates, OTIS Study

Hematopoietic and Lymphoid Cell Neoplasm | Acute Myeloid Leukemia | Hodgkin Lymphoma | Myelofibrosis | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Non-Hodgkin Lymphoma | Myelodysplastic Syndrome | Plasma Cell Myeloma | Aplastic Anemia | Myeloproliferative Neoplasm | Chronic Myelogenous Leukemia...

United States
Sidney Kimmel Cancer Center at Thomas Jefferson...

Active, not recruiting

Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Acute Myeloid Leukemia | Hodgkin Lymphoma | Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome | Plasma Cell Myeloma | Aplastic Anemia | Malignant Neoplasm | Myeloproliferative Neoplasm | Indolent Non-Hodgkin Lymphoma | Acute Myeloid Leukemia in Remission | Refractory Anemia | Refractory Anemia With... and other conditions

United States
Anjali Sharathkumar
Amgen

Active, not recruiting

TPO-Mimetic Use in Children for Hematopoietic Failure

Thrombocytopenia | Bone Marrow Failure Disorders | Aplastic Anemia | Refractory Cytopenia of Childhood | Myelodysplastic Syndrome(MDS)

United States
Cellphire Therapeutics, Inc.
Department of Health and Human Services

Terminated

Thrombosomes® in Bleeding Thrombocytopenic Patients Study

Myelodysplastic Syndromes | Myeloproliferative Disorders | Thrombocytopenia | Hematologic Malignancy | Bone Marrow Aplasia | Platelet Refractoriness

United States, Israel, Norway
Masonic Cancer Center, University of Minnesota

Active, not recruiting

Reduced Intensity (RIC) Conditioning And Transplantation of HLA-Haplo-HCT

Hematologic Malignancies

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
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
Masonic Cancer Center, University of Minnesota

Active, not recruiting

UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep

Follicular Lymphoma | Acute Myeloid Leukemia | Multiple Myeloma | Hodgkin Lymphoma | Lymphoplasmacytic Lymphoma | Acute Leukemia | Myelodysplastic Syndrome | Chronic Myelogenous Leukemia | Prolymphocytic Leukemia | Plasma Cell Leukemia | Bone Marrow Failure Syndromes | Burkitt's Lymphoma | Acute Lymphoblastic Leukemia... and other conditions

United States
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Completed

Engineered Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Acute Myeloid Leukemia | Recurrent Hodgkin Lymphoma | Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome | Chronic Myelomonocytic Leukemia | Acute Lymphoblastic Leukemia | Lymphoblastic Lymphoma | Myelodysplastic Syndrome | Plasma Cell Myeloma | Aplastic Anemia | Recurrent Non-Hodgkin... and other conditions

United States
Fondazione Italiana Sindromi Mielodisplastiche-ETS

Completed

Identification of Mechanism in the Erythroid Response in Patients With Myelodysplasia Undergoing Chelation Therapy (BIOFER12)

Myelofibrosis | Aplastic Anemia | Myelodysplasia

Italy
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Cystinosis Disease: BioCystinosis (BioCystinosis) (BioCystinosis)

Hypophosphatemia | Photophobia | Renal Fanconi Syndrome

Germany, India, Sri Lanka
Sidney Kimmel Cancer Center at Thomas Jefferson...
National Cancer Institute (NCI)

Completed

Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Malignancies

Follicular Lymphoma | Myeloid Leukemia | Polycythemia Vera | Hodgkin Lymphoma | Myelofibrosis | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Chronic Lymphocytic Leukemia | Secondary Acute Myeloid Leukemia | Non-Hodgkin Lymphoma | Myelodysplastic Syndrome | Plasma Cell Myeloma | Refractory... and other conditions

United States
Adrianna Vlachos, MD

Terminated

The Use of Trifluoperazine in Transfusion Dependent DBA (DBA)

Pure Red Cell Aplasia | Diamond Blackfan Anemia

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
Boston Children's Hospital

Completed

Pilot Study of Metformin for Patients With Fanconi Anemia

Fanconi Anemia

United States
Scripps Health

Completed

Nonmyeloablative Allogeneic Transplant (Mini-allo)

Melanoma | Sarcoma | Renal Cell Carcinoma | Breast Cancer | Multiple Myeloma | Hodgkin's Lymphoma | Non-Hodgkin's Lymphoma | Ovarian Cancer | Thymoma | Waldenstrom Macroglobulinemia | Mantle Cell Lymphoma | Chronic Lymphocytic Leukemia | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Chronic Myelogenous Leukemia and other conditions

United States
Scripps Health

Completed

Unrelated Donor Stem Cell Transplantation

Myelodysplastic Syndromes | Multiple Myeloma | Hodgkin's Lymphoma | Non-Hodgkin's Lymphoma | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Acute Myelogenous Leukemia | Chronic Myelogenous Leukemia | Small Lymphocytic Lymphoma | Paroxysmal Nocturnal Hemoglobinuria | Severe Aplastic Anemia | Myeloproliferative... and other conditions

United States
Sidney Kimmel Cancer Center at Thomas Jefferson...

Completed

A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies

Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | 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... and other conditions

United States
New York Medical College

Completed

Human Placental-Derived Stem Cell Transplantation (HPDSC)

Mucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditions

United States
University of Alabama at Birmingham

Completed

Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis

Lymphoma | Leukemia | Myelofibrosis | Myelodysplastic Syndrome | Severe Aplastic Anemia | Allogeneic Transplant

United States
Onconova Therapeutics, Inc.

Terminated

Controlled Study of Rigosertib Versus Physician's Choice of Treatment in MDS Patients After Failure of an HMA (INSPIRE)

Myelodysplastic Syndrome | MDS | Refractory Anemia With Excess Blasts | RAEB

United States, Spain, Canada, France, Australia, Switzerland, Israel, Japan, Belgium, India, Ireland, United Kingdom, Croatia, Czechia, Poland, Estonia, Germany, Italy, Austria, Hungary, Russian Federation, Sweden
Bellicum Pharmaceuticals

Active, not recruiting

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

Lymphoma, Non-Hodgkin | Myelodysplastic Syndromes | Acute Lymphoblastic Leukemia | Anemia, Aplastic | Primary Immune Deficiency Disorder | Cytopenia | Osteopetrosis | Leukemia, Acute Myeloid (AML), Child | Hemoglobinopathy in Children

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

Completed

Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or Myeloma

Chronic Myeloproliferative Disorders | Fanconi Anemia | Multiple Myeloma and Plasma Cell Neoplasm | Myelodysplastic/Myeloproliferative Neoplasms

United States
Celgene

Completed

A Study of Special Use Results Surveillance of Revlimid 5mg Capsules

Myelodysplastic Syndromes | Multiple Myeloma

Japan
Wake Forest University Health Sciences

Terminated

T-cell Depleted Alternative Donor Transplantation

Acute Myeloid Leukemia | Acute Lymphoblastic Leukemia | Chronic Myeloid Leukemia | Myelodysplastic Syndrome | Immune Deficiency | Lymphomas | Bone Marrow Failure | Osteopetrosis | Hemoglobinopathy

United States
Memorial Sloan Kettering Cancer Center
Pediatric Brain Tumor Consortium

Terminated

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

Fanconi Anemia | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML)

United States
Centre Leon Berard

Withdrawn

A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts (EVAADE)

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

France
Yale University

Withdrawn

CPX-351 in Patients Treated for Higher-risk Myelodysplastic Syndromes Experiencing Hypomethylating Agent Failure.

MDS

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

Completed

Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome

Severe Aplastic Anemia (SAA) | Myelodysplastic Syndrome (MDS) With Refractory Anemia (RA)

United States
Assistance Publique - Hôpitaux de Paris
EuroFancolen

Completed

Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor (FancoMob)

Fanconi Anemia

France
Masonic Cancer Center, University of Minnesota

Completed

Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia

Fanconi Anemia

United States
Masonic Cancer Center, University of Minnesota

Completed

TBI Dose De-escalation for Fanconi Anemia

Fanconi Anemia

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
Masonic Cancer Center, University of Minnesota

Completed

Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia

Fanconi Anemia

United States
University of California, San Francisco

Completed

Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

Sickle Cell Disease | Hemoglobinopathies | Thalassemia | Hematologic Malignancies | Immunodeficiencies | Fanconi's Anemia | Nonmalignant Diseases | Genetic Inborn Errors of Metabolism

United States
University of California, San Francisco

Completed

Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)

Sickle Cell Disease | Hemoglobinopathies | Thalassemia | Hematologic Malignancies | Immunodeficiencies | Fanconi's Anemia | Nonmalignant Diseases | Genetic Inborn Errors of Metabolism

United States

Search clinical trials for: Inherited Bone Marrow Failure Syndromes

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