Clinical Trials on Methemoglobinemia, Beta-globin Type

Total 37 results

Federal Research Institute of Pediatric Hematology...

Unknown

the Efficacy and Safety of Sugammadex in Children 0-2 Years Old

Anesthesia | Pediatric Cancer | Beta-Thalassemia | Hepatic Metastases | Pediatric Tumor | Pediatric Hepatoblastoma

Russian Federation
University of Thessaly

Completed

Resistance Exercise on Postprandial Hyperglycemia in Patients With B-thalassemia Exhibiting Resistance to Insulin

Diabetes Mellitus, Type 2 | Insulin Resistance | PreDiabetes | Beta-Thalassemia

Greece
Kyunghee University Medical Center
Sanofi; Korea University Guro Hospital; Hanyang University; The Catholic University... and other collaborators

Completed

Study To Evaluate Beta Cell Function and Glycemic Outcome by Intensive Insulin Therapy (KIIT)

Type 2 Diabetes Mellitus | Pancreatic Beta Cell Function | Glucotoxicity

Korea, Republic of
Finnish Institute for Health and Welfare
University of Turku; University of Helsinki; University of Eastern Finland; Valio...

Completed

Insulin-free Cow Milk Formula in Prevention of Type 1 Diabetes Associated Autoimmunity - FINDIA Pilot Study (FINDIA)

Beta-cell Autoimmunity
Rabin Medical Center
Kamada, Ltd.

Completed

Long Term Treatment Effect of the Safety, Tolerability and Efficacy of AAT in Type 1 Diabetes (AAT Extension)

Type 1 Diabetes | Beta Cell Preservation

Israel
Mitchell Cairo

Recruiting

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Hodgkin Lymphoma | Sickle Cell Disease | Acute Leukemia | Beta-Thalassemia | Non-hodgkin Lymphoma | Severe Aplastic Anemia | Diamond Blackfan Anemia | Amegakaryocytic Thrombocytopenia | Kostmann

United States
Melanoma and Skin Cancer Trials Limited
Melanoma Institute Australia; Cancer Council New South Wales

Active, not recruiting

Evaluation of Groin Lymphadenectomy Extent For Metastatic Melanoma (EAGLE FM)

Metastatic Melanoma to the Groin Lymph Nodes

United Kingdom, Slovenia, Australia, Brazil, Netherlands, Italy
Center for Vascular Pathology, Moscow

Enrolling by invitation

The Efficacy and Safety of Beta-blockers Drugs in Adults With Spinal Hemangioma

Spinal Tumor | Spinal Hemangioma | Beta Blocker Toxicity

Russian Federation
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
Wolfson Medical Center

Unknown

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

Patients With Thalassemia Intermedia, | Congenital Dyserythropoietic Anemia Type I
Vanderbilt University
National Cancer Institute (NCI)

Completed

Methemoglobinemia in Young Patients With Hematologic Cancer or Aplastic Anemia Treated With Dapsone

Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Multiple Myeloma and Plasma Cell Neoplasm | Nonmalignant Neoplasm | Lymphoproliferative Disorder | Myelodysplastic/Myeloproliferative Neoplasms | Methemoglobinemia

United States
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
University of Chicago
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Krabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)

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
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
Talaris Therapeutics Inc.
Duke University

Terminated

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Niemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...

United States
Rigshospitalet, Denmark

Unknown

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Carbohydrate Metabolism, Inborn Errors | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease Type V | VLCAD Deficiency | Glycogen Storage Disease Type III | Phosphoglycerate Kinase Deficiency | Neutral Lipid Storage Disease | Carnitine... and other conditions

Denmark
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
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
Masonic Cancer Center, University of Minnesota

Completed

Stem Cell Transplant for Inborn Errors of Metabolism

Niemann-Pick Disease | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | GM1 Gangliosidosis | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Tay Sachs Disease | Sandhoff Disease

United States
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
Fairview University Medical Center

Unknown

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Mucopolysaccharidosis I | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Pulmonary Complications | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Mannosidosis | I Cell Disease

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
Masonic Cancer Center, University of Minnesota

Terminated

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Sphingolipidoses | Niemann-Pick Disease, Type C | Krabbe Disease | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Hurler's Syndrome | Maroteaux-Lamy Syndrome | Wolman's Disease | Niemann-Pick Disease Type B

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
Fog Pharmaceuticals, Inc.

Recruiting

FOG-001 in Locally Advanced or Metastatic Solid Tumors

Cancer | Gastric Cancer | Colorectal Cancer | Solid Tumor | Metastatic Cancer | Adenomatous Polyposis Coli | Non-small Cell Lung Cancer | Non-small Cell Lung Cancer Metastatic | Non-small Cell Carcinoma | Locally Advanced Solid Tumor | Non-small Cell Lung Cancer Stage IIIB | Gastroesophageal-junction Cancer | Non-Small... and other conditions

United States
Joanne Kurtzberg, MD
The Marcus Foundation

Recruiting

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (DUOC-01)

Mucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo Mucopolysaccharidoses

United States
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
University of Pittsburgh

Recruiting

Longitudinal Study of Neurodegenerative Disorders

Lysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditions

United States
Institut National de la Santé Et de la Recherche...

Not yet recruiting

Conception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity and Inflammation (ATRACTion)

Autoimmune Diseases | Systemic Lupus Erythematosus | Primary Immunodeficiency | Autoimmune Hepatitis | Juvenile Idiopathic Arthritis | IBD | Autoimmune Rheumatologic Disease | Autoimmune Diabetes | APECED | IPEX | Hemophagocytic Lymphohistiocytoses | Autoimmune Lymphoproliferative Syndrome | Autoimmune Thrombocytopenia and other conditions
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
Centre Hospitalier Régional de la Citadelle
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Universitaire... 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

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
National Cancer Institute (NCI)

Completed

Temsirolimus and Bevacizumab in Treating Patients With Advanced Endometrial, Ovarian, Liver, Carcinoid, or Islet Cell Cancer

Uterine Carcinosarcoma | Recurrent Fallopian Tube Carcinoma | Recurrent Ovarian Carcinoma | Recurrent Primary Peritoneal Carcinoma | Recurrent Uterine Corpus Carcinoma | Ovarian Endometrioid Adenocarcinoma | Ovarian Seromucinous Carcinoma | Endometrial Serous Adenocarcinoma | Lung Carcinoid Tumor | Metastatic... and other conditions

United States, Canada
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

Clinical Trials on Methemoglobinemia, Beta-globin Type

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