Clinical Trials

17 trials
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NCT05687474 2025-08-12

BabyDetect

Centre Hospitalier Universitaire de Liege

Completed
6,824 enrolled
Classical hematologyEndocrineHead & NeckMyeloidOther solid neoplasm

Ataxia with vitamin E deficiency, Beta ketothiolase deficiency, Carnitine palmitoyl transferase 1A deficiency, Carnitine palmitoyl transferase 2 deficiency, dopamine beta hydroxylase deficiency, Malonic aciduria, Nephrosis, congenital, Cholestasis, progressive familial intrahepatic 1, Medium chain acyl CoA dehydrogenase deficiency, Pyridoxine-dependent epilepsy, Neonatal-onset citrullinemia type 2, Systemic carnitine deficiency, Glut1 Deficiency Syndrome, Glutaric Acidemia I, Timothy syndrome, Brown-Vialetto-Van Laere syndrome, Methylmalonic acidemia, Aromatic amino acid decarboxylase deficiency, Succinyl-CoA:3-oxoacid CoA transferase deficiency, Segawa syndrome, autosomal recessive, Neutropenia, Severe Congenital, Autosomal Recessive 3, Basal ganglia disease, biotin-responsive, Sucrase-isomaltase deficiency, congenital, Acidemia, isovaleric, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, HHH syndrome, Glucose-Galactose Malabsorption, Hypophosphatasia, Infantile, Dystonia, Dopa-Responsive, due to Sepiapterin Reductase Deficiency, Carnitine-Acylcarnitine Translocase Deficiency, Hypermethioninemia, Cardiomyopathy, Familial Hypertrophic, 4, Pyridoxamine 5-Prime-Phosphate Oxidase Deficiency, Phosphoglycerate Dehydrogenase Deficiency, Trifunctional Protein Deficiency With Myopathy And Neuropathy, Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of, Phosphoserine Aminotransferase Deficiency, Inflammatory Bowel Disease 25, Autosomal Recessive, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency, Neurodegeneration Due To Cerebral Folate Transport Deficiency, Glycogen Storage Disease XIV, Combined Pituitary Hormone Deficiency, Shwachman-Diamond Syndrome, Polymorphic Catecholaminergic Ventricular Tachycardia, Adrenal Hyperplasia, Congenital, Adrenoleukodystrophy, Anemia, Sickle Cell, Charcot-Marie-Tooth Disease, Chediak-Higashi Syndrome, Hemophilia B, Congenital Hypothyroidism, Crigler-Najjar Syndrome, Cystic Fibrosis, Cystinosis, Diabetes Mellitus, Type 2, Fanconi Syndrome, Fanconi Anemia, Fructose Intolerance, Galactosemias, Gaucher Disease, Glucosephosphate Dehydrogenase Deficiency, Glycogen Storage Disease, Glycogen Storage Disease Type II, Granulomatous Disease, Chronic, Hemophilia A, Hepatolenticular Degeneration, Homocystinuria, Hyperoxaluria, Primary, Menkes Kinky Hair Syndrome, Leukodystrophy, Metachromatic, Mucopolysaccharidosis I, Hyperlipoproteinemia Type I, Maple Syrup Urine Disease, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI, Nephritis, Hereditary, Neuronal Ceroid-Lipofuscinoses, Phenylketonurias, Pseudohypoaldosteronism, Retinoblastoma, Riboflavin Deficiency, Vitamin B 12 Deficiency, Wiskott-Aldrich Syndrome, Wolman Disease, Fructose-1,6-Diphosphatase Deficiency, Severe Combined Immunodeficiency, Mucopolysaccharidosis II, Mucopolysaccharidosis VII, Stiff-Person Syndrome, alpha-Thalassemia, Diabetes Insipidus, Nephrogenic, Smith-Lemli-Opitz Syndrome, alpha 1-Antitrypsin Deficiency, Hyperglycinemia, Nonketotic, Citrullinemia, Hyperargininemia, Ornithine Carbamoyltransferase Deficiency Disease, Carbamoyl-Phosphate Synthase I Deficiency Disease, Tyrosinemias, Myasthenic Syndromes, Congenital, Biotinidase Deficiency, Holocarboxylase Synthetase Deficiency, Anemia, Diamond-Blackfan, Jervell-Lange Nielsen Syndrome, Congenital Hyperinsulinism, Andersen Syndrome, Lymphohistiocytosis, Hemophagocytic, Familial Hypophosphatemic Rickets, Aspartylglucosaminuria, Propionic Acidemia, Argininosuccinic Aciduria

NCT00053989 2020-02-10

NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders

Roswell Park Cancer Institute

Phase 2 Completed
41 enrolled 13 charts
Classical hematologyLymphoidMyeloidOther hematologic neoplasmPlasma cell

Congenital Abnormalities, Anemia, Aplastic, Anemia, Refractory, Anemia, Refractory, with Excess of Blasts, Burkitt Lymphoma, Fanconi Anemia, Hodgkin Disease, Leukemia, Lymphoma, Lymphoma, Follicular, Waldenstrom Macroglobulinemia, Multiple Myeloma, Myelodysplastic Syndromes, Myeloproliferative Disorders, Recurrence, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Myeloid, Accelerated Phase, Leukemia, Myeloid, Chronic-Phase, Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Chronic, Lymphoma, B-Cell, Marginal Zone, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Neoplasms, Plasma Cell, Myelodysplastic-Myeloproliferative Diseases, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Primary Myelofibrosis

NCT00856388 2019-11-13

Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders

Roswell Park Cancer Institute

Phase NA Completed
62 enrolled 14 charts
Classical hematologyDermatologicLymphoidMyeloidOther hematologic neoplasmPlasma cellSarcoma

Pdgfra-Associated Chronic Eosinophilic Leukemia, Congenital Abnormalities, Anemia, Aplastic, Burkitt Lymphoma, Fanconi Anemia, Hemoglobinuria, Paroxysmal, Hodgkin Disease, Immunoblastic Lymphadenopathy, Lymphoma, Follicular, Lymphoma, Non-Hodgkin, Waldenstrom Macroglobulinemia, Multiple Myeloma, Mycosis Fungoides, Myeloproliferative Disorders, Recurrence, Sezary Syndrome, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Myeloid, Accelerated Phase, Leukemia, Myeloid, Chronic-Phase, Leukemia, Neutrophilic, Chronic, Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Chronic, Lymphoma, Large-Cell, Immunoblastic, Lymphoma, Large B-Cell, Diffuse, Lymphoma, T-Cell, Cutaneous, Lymphoma, Large-Cell, Anaplastic, Lymphoma, B-Cell, Marginal Zone, Lymphoma, Mantle-Cell, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, Lymphoma, Extranodal NK-T-Cell, Leukemia, Myelomonocytic, Juvenile, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Dendritic Cell Sarcoma, Interdigitating, Primary Myelofibrosis

NCT00305708 2012-11-12

Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission

University of California, San Francisco

Phase 1/2 Completed
40 enrolled
Classical hematologyOther hematologic neoplasm

Congenital amegakaryocytic thrombocytopenia, Neutropenia, Severe Congenital, Autosomal Recessive 3, Fanconi Anemia, Leukemia, Thrombocytopenia, Leukemia, Myeloid, Chronic-Phase, Anemia, Diamond-Blackfan

NCT00005892 2005-06-24

Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia

Office of Rare Diseases (ORD)

Phase NA Completed
Classical hematologyLymphoidMyeloidOther hematologic neoplasm

Anemia, Aplastic, Fanconi Anemia, Hematologic Diseases, Leukemia, Myelodysplastic Syndromes, Leukemia, Biphenotypic, Acute, Leukemia, Myeloid, Acute, Hematologic Neoplasms, Rare Diseases, Precursor Cell Lymphoblastic Leukemia-Lymphoma