Search clinical trials for: Hereditary Hemolytic Anemias

Total 4526 results

Massachusetts General Hospital
Agios Pharmaceuticals, Inc.

Recruiting

Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Myeloproliferative Neoplasm | Myelodysplastic/Myeloproliferative Neoplasm | Clonal Cytopenia of Undetermined Significance | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency Anemia | Hereditary Hemolytic Anemia | Clonal Myeloid Neoplasm | Other... and other conditions

United States
Sohag University
Assiut University

Active, not recruiting

Parvovirus B19 Infection in Hereditary Hemolytic Anemias Patients

Parvovirus B19 Infection

Egypt
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
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders (DANNIgene)

Hemophilia A | Hemophilia B | Cystic Fibrosis | Sickle Cell Disease | Muscular Dystrophy, Duchenne | Fragile X Syndrome | Huntington Disease | Myotonic Dystrophy | Autosomal Recessive Polycystic Kidney Disease | Neurofibromatosis-Noonan Syndrome | Muscular Dystrophy, Becker | Invasive PreNatal Diagnosis in a... and other conditions

France
The 923rd Hospital of Joint Logistics Support Force...
Guangzhou Reforgene Medicine Co., Ltd.

Recruiting

Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

Hemoglobinopathies | Hereditary Diseases | Alpha Thalassemia Hemoglobin H Constant Spring

China
National Human Genome Research Institute (NHGRI)

Recruiting

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Metabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditions

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
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
Sanguine Biosciences

Recruiting

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

Heart Diseases | Stroke | Lymphoma | Multiple Sclerosis | Kidney Diseases | Diabetes | Leukemia | Age-Related Macular Degeneration | Cancer | Arthritis | Schizophrenia | Thyroid Diseases | Parkinson Disease | Hepatitis | Vasculitis | Psoriasis | Asthma | Amyloidosis | Fibromyalgia | Graves Disease | Alzheimer Disease | Celiac Disease | Cirr... and other conditions

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
Assistance Publique - Hôpitaux de Paris

Recruiting

National Exhaustive Cohort of Hereditary Stomatocytoses and Other Channelopathies Affecting the Red Blood Cell (COHSTO)

Stomatocytosis

France
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
University of Milano Bicocca
Comitato Maria Letizia Verga,Italy

Recruiting

Precision Exercise in Children With Malignant Hemopathies (SportTherapy)

Lymphoma, Non-Hodgkin | Leukemia, Myeloid, Acute | Leukemia, B-cell | Adrenoleukodystrophy | Lymphoma, Hodgkin | Thalassemia Major | Leukemia, T Cell | Drepanocytosis

Italy
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
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
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
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
Insel Gruppe AG, University Hospital Bern
Swiss National Science Foundation; Mach Gaensslen Foundation; Baxalta Innovations...

Recruiting

Genotype and Phenotype Correlation in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome) (TTP registry)

Thrombotic Thrombocytopenic Purpura | Congenital Thrombotic Thrombocytopenic Purpura | Familial Thrombotic Thrombocytopenic Purpura | Thrombotic Thrombocytopenic Purpura, Congenital | Upshaw-Schulman Syndrome

United States, Austria, Czechia, Germany, Japan, Norway, Switzerland
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)

Completed

Validating Pain Scales in Children and Young Adults

Leukemia | Solid Tumor | Sickle Cell Disease | Neurofibromatosis

United States
Sanguine Biosciences

Completed

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

Depression | Melanoma | Epilepsy | Lymphoma | Multiple Sclerosis | Kidney Cancer | Cervical Cancer | Diabetes Mellitus | Leukemia | Breast Cancer | Chronic Kidney Diseases | Chronic Obstructive Pulmonary Disease | Parkinson's Disease | Systemic Lupus Erythematosus | Multiple Myeloma | Hepatitis B | Insomnia | Colorectal Cancer | HIV/AID... and other conditions

United States
Hospices Civils de Lyon

Completed

Hereditary Spherocytosis and Vascular Function (VASCUSPHERO)

Hereditary Spherocytosis

France
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Early Check: Expanded Screening in Newborns

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions

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

Active, not recruiting

Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

Sickle Cell Disease | Congenital Hemolytic Anemia

United States
Boston Children's Hospital

Completed

Senicapoc and Dehydrated Stomatocytosis

Dehydrated Hereditary Stomatocytosis

United States
Masonic Cancer Center, University of Minnesota

Terminated

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases

Sickle Cell Disease | Thalassemia | Cerebral Adrenoleukodystrophy | Inherited Metabolic Disorders | High Risk Hematologic Disorders

United States
ApoPharma

Terminated

Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias

Iron Overload | Sickle Cell Disease | Other Anemias

United States, United Kingdom, Egypt, Canada, Saudi Arabia
National Heart, Lung, and Blood Institute (NHLBI)

Active, not recruiting

Haploidentical PBMC Transplant for Severe Congenital Anemias

Sickle Cell Anemia

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
ApoPharma

Terminated

Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias (FIRST)

Iron Overload | Sickle Cell Disease | Other Anemias

United States, Turkey, United Kingdom, Egypt, Saudi Arabia, Brazil, Canada, Tunisia
University of Milano Bicocca

Completed

Frequency of COVID-19 Antibodies in Patients With Hereditary Hematological Diseases (ThalaCoV)

Covid19 | Thalassemia Major

Italy
Abfero Pharmaceuticals, Inc

Withdrawn

SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias

Iron Overload | Beta-Thalassemia

Thailand, Lebanon, Turkey, Canada
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
Novartis Pharmaceuticals

Completed

Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

Myelodysplastic Syndromes | Fanconi Syndrome | Anemia, Aplastic | Beta-thalassemia | Anemia, Diamond-Blackfan

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
Novartis Pharmaceuticals

Completed

An Epidemiological Study to Assess Iron Overload Using MRI in Patients With Transfusional Siderosis (TIMES Study)

Thalassemia, Non-transfusional-dependent Thalassemia (NTDT), Myeloplastic Dysplasia (MDS), Other Anemia

Australia
Children's Mercy Hospital Kansas City

Withdrawn

Single Incision Versus Standard Laparoscopic Splenectomy

Idiopathic Thrombocytopenic Purpura | Hereditary Spherocytosis

United States
Memorial Sloan Kettering Cancer Center

Completed

Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major

Beta Thalassemia Major | Congenital Anemias

United States
Children's Hospital Los Angeles

Completed

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

Granuloma | Anemia | Thrombocytopenia | Neutropenia | Sickle Cell Disease | Thalassemia | Niemann-Pick Disease | Wiskott-Aldrich Syndrome | Osteopetrosis | Fucosidosis | Chediak Higashi Syndrome | Hurler Disease

United States
University of British Columbia

Unknown

Utility of Fibroscan in Estimating Hepatic Iron Concentration

Hereditary Anemias

Canada
University Health Network, Toronto

Completed

MR Imaging of Diffuse Myocardial Fibrosis in Transfusion-Dependent Anemias (MAFIO)

Thalassemia Major | Chronic Blood Transfusion Related Iron Overload

Canada
University of Texas Southwestern Medical Center

Terminated

The Dallas Hereditary Spherocytosis Cohort Study

Hereditary Spherocytosis
Novartis Pharmaceuticals

Completed

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

Myelofibrosis | Sickle Cell Disease | Thalassemia | Diamond Blackfan Anemia

United States, Netherlands, Taiwan, Thailand, Italy, Germany, Spain, Canada, Belgium, Greece, Turkey, United Kingdom
Novartis Pharmaceuticals

Completed

Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

Anemia | Hemosiderosis

United States, Germany, Belgium, United Kingdom, Italy, Canada, France
Wolfson Medical Center

Unknown

GDF 15 in Sickle Cell Disease and Hereditary Spherocytosis (GDF 15)

Patients With Thalassemia Intermedia, | Congenital Dyserythropoietic Anemia Type I
Wolfson Medical Center

Unknown

Hemolysis in Patients With Hereditary Spherocytosis (HS)

Hemolysis | Hereditary

Israel
University of Utah
University of Witwatersrand, South Africa

Completed

A Novel Mutation of the Spectrin Gene

Hereditary Elliptocytosis (HE) | Hereditary Pyropoikilocytosis (HPP)

United States
National Center for Research Resources (NCRR)
University of California, Los Angeles

Completed

Stem Cell Transplantation (SCT) for Genetic Diseases

Thrombocytopenia | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Fanconi's Anemia | Thalassemia Major | Pure Red-Cell Aplasia

United States
Eye & ENT Hospital of Fudan University
The Eye Hospital of Wenzhou Medical University

Not yet recruiting

Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma

Retinoblastoma | Refractory
University of Cologne
The Clinical Trials Centre Cologne; ESCRS (European Society of Cataract and...

Not yet recruiting

Trial Comparing Cataract Surgery With Triple-DMEK in Patients With Cataract and Fuchs Endothelial Corneal Dystrophy

Cataract Surgery | Cataract and Fuchs Endothelial Corneal Dystrophy

Denmark, Germany, Netherlands, Spain

Search clinical trials for: Hereditary Hemolytic Anemias

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