Clinical Trials on Beta Thalassemia

Total 569 results

Travere Therapeutics, Inc.

Recruiting

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment (HARMONY)

Homocystinuria

United States
TriVirum, Inc.

Completed

Clinical Performance of the AFGen1 Device Over a 7-day Period

Atrial Fibrillation | Arrhythmias, Cardiac | Paroxysmal Atrial Fibrillation | Afib | Irregular Heart Beat | Arrhythmia Atrial | Arrhythmias Paroxysmal

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

Completed

Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary Hypertension

Hypertension, Pulmonary | Thalassemia

United States
Assistance Publique - Hôpitaux de Paris
Novartis

Completed

Multicenter Observational Study on Myocardial Iron Overload in 3 Multitransfused Populations (SUFEMYO)

Sickle Cell Disease | Thalassemia | Myelodysplasia

France
Phoenix Children's Hospital

Completed

A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT

Myelodysplastic Syndromes | Vitamin D Deficiency | Pediatric Cancer | Aplastic Anemia | Stem Cell Transplant Complications | Sickle Cell Anemia in Children | Thalassemia in Children | Blood Disorder | Pediatric Acute Myeloid Leukemia | Pediatric Acute Lymphoid Leukemia

United States
The Methodist Hospital Research Institute
M.D. Anderson Cancer Center; National Institute of Allergy and Infectious Diseases... and other collaborators

Recruiting

Dynamics of Colonization and Infection by Multidrug-Resistant Pathogens in Immunocompromised and Critically Ill Patients (DYNAMITE)

Antibiotic Resistant Infection | Clostridium Difficile | Antimicrobial Drug Resistance | Carbapenem-Resistant Enterobacteriaceae Infection | Vancomycin Resistant Enterococci Infection | Vancomycin-Resistant Enterococcal Infection | Carbapenem Resistant Bacterial Infection | Extended Spectrum Beta-Lactamase...

United States
Tianjin Medical University General Hospital

Not yet recruiting

Propranolol on Post Stroke Immune Status and Infection

Cardiovascular Diseases | Stroke | Cerebrovascular Disorders | Brain Diseases | Central Nervous System Diseases | Nervous System Diseases | Ischemic Stroke | Molecular Mechanisms of Pharmacological Action | Physiological Effects of Drugs | Propranolol | Immunologic Factors | Beta Blocker

China
Novartis Pharmaceuticals

Completed

This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.

Myelodysplastic Syndrome | Thalassemia

Russian Federation
LMU Klinikum
Seventh Framework Programme; NBIA Alliance

Recruiting

TIRCON International NBIA Registry (TIRCON)

Neurodegeneration With Brain Iron Accumulation (NBIA) | Pantothenate Kinase-associated Neurodegeneration (PKAN) | Aceruloplasminemia | Beta-Propeller Protein-Associated Neurodegeneration (BPAN) | Mitochondrial Membrane Protein Associated Neurodegeneration (MPAN) | Fatty Acid Hydroxylase-associated... and other conditions

Canada, Czechia, Germany, Italy, Netherlands, Poland, Serbia, Spain
Universitas Padjadjaran
Hasan Sadikin General Hospital

Completed

Immunomodulatory Vitamin D in Thalassemia (ThalTB)

Vitamin D Deficiency | Tuberculosis | Thalassemia

Indonesia
Baqiyatallah Medical Sciences University
Guilan University of Medical Sciences; Baqiyatallah Research Center for Gastroenterology... and other collaborators

Completed

Pegasys® Plus Ribavirin in Thalassemic Patients With Hepatitis C Virus Infection

Hepatitis C | Thalassemia

Iran, Islamic Republic of
Aeglea Biotherapeutics

Terminated

A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

Homocystinuria Due to Cystathionine Beta-Synthase Deficiency

United States, United Kingdom, Australia
Ala'a Sharara
Gilead Sciences

Completed

Sofosbuvir/Ledipasvir for Hepatitis C Genotype 1-6 in Patients With Transfusion-Dependent Thalassemia: An Open Label Trial

Hepatitis C | Thalassemia

Lebanon
HealthCore-NERI
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Evaluating the Safety of Two Medications to Treat Hepatitis C in People With Thalassemia (The HepC Study) (HepC)

Hepatitis C | Thalassemia

United States, United Kingdom, Canada
UCSF Benioff Children's Hospital Oakland
Food and Drug Administration (FDA)

Completed

Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension

Sickle Cell Disease | Pulmonary Hypertension | Thalassemia

United States
University of Southern California
Kaiser Permanente

Completed

Genetics of Beta Cell Failure in Mexican Americans

Obesity | Physical Activity | Insulin Resistance | Genetics | Air Pollution | Gestational Diabetes | Nutrition | Diabetes Type 2 | Adipokines | Beta Cell Function
Stanford University
University of Alabama at Birmingham; University of Minnesota

Completed

Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs

Sickle Cell Disease | Thalassemia | Diamond-Blackfan Anemia

United States
IRCCS Policlinico S. Donato

Terminated

Use of Ritmonutra in Subjects Affected by Supraventricular Ectopic Beats Without Structural Heart Disease

Arrhythmia

Italy
Medical College of Wisconsin
National Heart, Lung, and Blood Institute (NHLBI); University of Wisconsin,... and other collaborators

Recruiting

Fetal Electrophysiologic Abnormalities in High-Risk Pregnancies Associated With Fetal Demise

Sudden Infant Death | Congenital Heart Disease | Pregnancy Loss | Gastroschisis | Fetal Death | Brugada Syndrome | High Risk Pregnancy | Stillbirth | Long QT Syndrome | Fetal Demise | Birth Defect | Twin Monochorionic Monoamniotic Placenta | Twin Twin Transfusion Syndrome | Fetal Hydrops | Fetal Arrhythmia | Intrauterine... and other conditions

United States
UCSF Benioff Children's Hospital Oakland
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Medical... and other collaborators

Completed

Multi-Center Study of Iron Overload: Pilot Study (MCSIO)

Sickle Cell Disease | Thalassemia | Diamond-Blackfan Anemia

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

Completed

Deferoxamine for the Treatment of Hemochromatosis

Diabetes Mellitus | Heart Disease | Thalassemia | Hemochromatosis

United States
Duke University

Completed

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Hemoglobinopathies | Thalassemia | Lysosomal Storage Disease | Immunodeficiencies | Inborn Errors of Metabolism | Sickle Cell | Non Malignant Disorders | Congenital Marrow Failures

United States
University of Valencia

Recruiting

CSF Protein Markers as Prognostic Indicators of the Response to CSF Shunt in Normotensive Hydrocephalus

Cerebrospinal Fluid Levels of Amyloid Beta 1-42, H-tau and Phospho-tau Proteins | Cognitive Impairment and Its Evolution After Hydrocephalus Treatment | Motor Impairment and Its Evolution After Hydrocephalus Treatment | Ventricular Size and Its Evolution After Implantation of a Ventriculoperitoneal...

Spain
University Hospital of North Norway
Norwegian Extra Foundation for Health and Rehabilitation

Completed

Emotional and Cognitive Determinants of Post-stroke Fatigue

Stroke | Quality of Life | Fatigue | Cognitive Impairment | Post-stroke Depression | Neuropsychology | Post-Stroke Fatigue | Meta-cognition

Norway
Chelsea Therapeutics

Completed

Open-Label Clinical Study of Droxidopa in Patients With Neurogenic Orthostatic Hypotension (NOH) (NOH303)

Multiple System Atrophy | Neurogenic Orthostatic Hypotension | Dopamine Beta Hydroxylase Deficiency | Non-Diabetic Autonomic Neuropathy

United States, Australia, Canada, New Zealand
Passage Bio, Inc.

Active, not recruiting

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis (Imagine-1)

GM1 Gangliosidosis | GM1 Gangliosidosis, Type I | GM1 Gangliosidosis, Type 2 | Beta-Galactosidase-1 (GLB1) Deficiency

United States, Canada, United Kingdom, Turkey, Brazil
HaEmek Medical Center, Israel

Completed

Clinical Diagnosis of Teenagers Admitted to Pediatric Departments

Anorexia | Sickle Cell Anemia | Thalassemia | Chronic Renal Failure | Suicide Attempt

Israel
Novartis Pharmaceuticals

Recruiting

CINC424A2X01B Rollover Protocol

Acute Myeloid Leukemia | Primary Myelofibrosis | Polycythemia Vera | Thalassemia | Graft Versus Host Disease

Korea, Republic of, Italy, Belgium, Japan, Germany, Australia, Bulgaria, Chile, China, Denmark, Greece, Hungary, Israel, Lebanon, Poland, Portugal, Russian Federation, Slovakia, South Africa, Spain, Sweden, Thailand, India, Mexico, Turkey, Fran...
Genzyme, a Sanofi Company

Recruiting

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

Gaucher Disease | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease

Hungary, Croatia, United States, Australia, Belgium, Brazil, Bulgaria, Canada, Denmark, Germany, Greece, Hong Kong, India, Indonesia, Italy, Jordan, Korea, Republic of, Kuwait, Lebanon, Malaysia, Pakistan, Philippines, Poland, Saudi Arabia and more
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
UCSF Benioff Children's Hospital Oakland
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Medical... and other collaborators

Completed

Multi-Center Study of Iron Overload: Survey Study (MCSIO) (MCSIO)

Sickle Cell Disease | Thalassemia | Diamond-Blackfan Anemia

United States, United Kingdom, Germany
Chelsea Therapeutics

Terminated

A Clinical Study of Patients With Symptomatic NOH to Assess Sustained Effects of Droxidopa Therapy

Parkinson's Disease | Pure Autonomic Failure | Dopamine Beta Hydroxylase Deficiency | Symptomatic Neurogenic Orthostatic Hypotension | Multiple Systems Atrophy

United States
Northwestern University
GlaxoSmithKline; National Comprehensive Cancer Network; Robert H. Lurie Cancer...

Completed

Temozolomide and Pazopanib Hydrochloride in Treating Patients With Advanced Pancreatic Neuroendocrine Tumors That Cannot Be Removed By Surgery

Recurrent Islet Cell Carcinoma | Pancreatic Alpha Cell Carcinoma | Pancreatic Beta Islet Cell Carcinoma | Pancreatic Delta Cell Carcinoma | Pancreatic G-cell Carcinoma

United States
Genzyme, a Sanofi Company

Completed

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

Gaucher Disease, Type 1 | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic Form

United States, United Kingdom, Spain, Italy, Brazil, Canada, Poland
Genzyme, a Sanofi Company

Completed

A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Cerebroside Lipidosis Syndrome | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease Type I | Clucocerebrosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic Form

United States
AC Immune SA
Worldwide Clinical Trials

Recruiting

A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)

Alzheimer's Disease | Prodromal Alzheimer's Disease | Amyloid Plaque | Beta-Amyloid | Alzheimer's Disease in Down Syndrome

United States, Spain, United Kingdom
Genzyme, a Sanofi Company

Completed

A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

Gaucher Disease, Type 1 | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic Form

United States, Israel, Mexico, Russian Federation, Argentina, Italy
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
University of California, San Francisco
Thrasher Research Fund

Completed

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

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

United States
Great Ormond Street Hospital for Children NHS Foundation...
National Institute for Health Research, United Kingdom; The Freya Foundation

Recruiting

Natural History Study of Pyruvate Dehydrogenase Deficiency

Pyruvate Dehydrogenase Complex Deficiency | Pyruvate Dehydrogenase E1 Alpha Deficiency | Pyruvate Dehydrogenase E1-Beta Deficiency | Pyruvate Dehydrogenase E2 Deficiency | Pyruvate Dehydrogenase Phosphatase Deficiency

United Kingdom
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
Masonic Cancer Center, University of Minnesota
National Marrow Donor Program

Completed

Stem Cell Transplant for Hemoglobinopathy

Sickle Cell Disease | Thalassemia | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Shwachman-Diamond Syndrome

United States
National Medical Research Center for Rehabilitation...
Pirogov Russian National Research Medical University; DNKOM LLC; The Orenburg...

Recruiting

Study of Efficacy and Safety of the Plasmapheresis Method With Albumin Compensation Compared With the Plasmapheresis Method Without Albumin Compensation for Aging Biomarkers Correction in Men and Women Aged 40 to 55 Years Old

Cardiovascular Diseases | Nervous System Diseases | Urologic Diseases | Diabetes | Neuromuscular Diseases | Hypercholesterolemia | Gout | Hyperbilirubinemia | Hyper IgE Syndrome | Hyper-LDL-cholesterolemia | Hyper Beta Lipoproteinemia | Hyper Eosinophilic Syndrome

Russian Federation
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
West Kazakhstan Medical University

Recruiting

Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan

Ornithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditions

Kazakhstan
Masonic Cancer Center, University of Minnesota

Recruiting

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

Myelodysplastic Syndromes | Fanconi Anemia | Dyskeratosis Congenita | Severe Aplastic Anemia | Bone Marrow Failure | T Cell Receptor Alpha/Beta Depletion | Telomere Biology Disorder

United States
Cardiologs Technologies

Completed

Electrocardiographic Diagnostic Performance of the Apple Watch Augmented With an Artificial Intelligence Algorithm

Atrial Fibrillation | Bradycardia | Tachycardia | Premature Ventricular Contraction | Premature Supraventricular Beat

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
Hospital Universitari Vall d'Hebron Research Institute
ERN-EuroBloodNet (European Reference Network on Rare Hematological Diseases

Recruiting

European Rare Blood Disorders Platform (ENROL) (ENROL)

Leukemia | Anemia | Iron Metabolism Disorders | Anemia, Sickle Cell | Thalassemia | Bleeding Disorder | Myeloma | Lymphoid Neoplasm | Blood Cancer | Bone Marrow Failure | Red Cell Membrane and Enzyme Abnormalities | Myeloma, Malignant

Spain
American Thrombosis and Hemostasis Network
Novo Nordisk A/S; Sanofi; Pfizer; Genentech, Inc.; CSL Behring; Hemophilia of Georgia... and other collaborators

Recruiting

ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

Hemophilia | Thrombosis | Thrombophilia | Sickle Cell Disease | Thalassemia | Bleeding Disorder | Von Willebrand Diseases | Factor IX Deficiency | Connective Tissue Disorder | Factor VIII Deficiency | Hematologic Disorder | Rare Bleeding Disorder | Platelet Disorder

United States

Clinical Trials on Beta Thalassemia

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