Clinical Trials on Leaky SCID
Total 1463 results
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Tisento TherapeuticsChildren's Hospital of PhiladelphiaTerminated
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Enzyvant Therapeutics GmBHCompletedFarber Disease | Farber's Disease | Farber Lipogranulomatosis | Acid Ceramidase Deficiency | Ceramidase Deficiency | N-Laurylsphingosine Deacylase Deficiency | ASAH1 MutationUnited States, Egypt, Canada, Italy, Turkey, Argentina, Germany, India, Sweden
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Genzyme, a Sanofi CompanyCompletedPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2United States, France, Netherlands
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National Taiwan University HospitalUnknownAlkaline Chemical Burn Of Cornea And Conjunctival Sac | Acid Chemical Burn Of Cornea And Conjunctival Sac | Benign Mucous Membrane Pemphigoid With Ocular InvolvementTaiwan
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AstraZenecaRecruitingUsing NCID Database to Assess the Safety of Olaparib in Chinese Patients With Ovarian Cancer by Examining the Incidence, Seriousness, and Severity of All AEChina
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Benha UniversityCompletedTransient Tachypnea of Newborn and Fetal AcidosisEgypt
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Genzyme, a Sanofi CompanyCompletedHuman Acid Sphingomyelinase DeficiencyUnited Kingdom, United States
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Assistance Publique - Hôpitaux de ParisCompletedOculocerebrorenal SyndromeFrance
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Tisento TherapeuticsNot yet recruitingMitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)United States, Australia, Italy, Germany, Canada, United Kingdom
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Chung-Hsing WangSanofiNot yet recruiting
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SanofiRecruitingAcid Sphingomyelinase Deficiency (ASMD)France
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Queen's University, BelfastUniversity of SussexUnknownRetinitis Pigmentosa | Low Vision | Albinism | Stargardt Disease 1 | Stargardt Disease 3 | Stargardt Disease 4United Kingdom
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National Heart, Lung, and Blood Institute (NHLBI)CompletedMyocardial Infarction | Heart Diseases | Cardiovascular Diseases | Cerebrovascular Disorders | Peripheral Vascular Diseases | Atherosclerosis | Hyperhomocysteinemia
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Genzyme, a Sanofi CompanyCompletedPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2Netherlands
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Genzyme, a Sanofi CompanyCompletedPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2France
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Karunesh GangulyNational Institute on Deafness and Other Communication Disorders (NIDCD)RecruitingStroke | Multiple Sclerosis | Muscular Dystrophies | ALS | SCI - Spinal Cord InjuryUnited States
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Novartis PharmaceuticalsCompletedSickle Cell Disease (SCD)United Kingdom, Spain, Greece, Brazil, Turkey, United States, France, Netherlands, Lebanon, Panama, Ireland
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National Human Genome Research Institute (NHGRI)RecruitingHermansky-Pudlak Syndrome (HPS)United States
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Ultragenyx Pharmaceutical IncTerminatedWilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IASpain, United Kingdom, Canada, United States, Brazil
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SanofiRecruitingAcid SphingoMyelinase Deficiency | Gaucher Disease, SplenomegalyFrance
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Hennepin Healthcare Research InstituteUniversity of Minnesota; Regions HospitalCompletedSmoking | Inflammation | Surgery | Surgery--Complications | Wound Infection | Oxidative Stress | Wound | Mandible Fracture | Mandibular Fractures | Vitamin C Deficiency | Ascorbic Acid Deficiency | Wound Complication | Wound of Skin | Wound Dehiscence | Mandible Open Fracture | Mandible Closed FractureUnited States
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University Hospital, RouenUnknownObesity | Acute Respiratory Failure | Hypercapnic Respiratory Failure | Apnea, Obstructive | Hypoventilation Syndrome | Respiratory Acidosis
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Skane University HospitalLund UniversityRecruitingAlzheimer Disease | Frontotemporal Degeneration | Mild Cognitive Impairment | Lewy Body Disease | Vascular Dementia | SCD | Mild DementiaSweden
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Skane University HospitalLund UniversityRecruitingAlzheimer Disease | Frontotemporal Degeneration | Mild Cognitive Impairment | Lewy Body Disease | Vascular Dementia | SCD | Mild DementiaSweden
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West Kazakhstan Medical UniversityRecruitingOrnithine 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 conditionsKazakhstan
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Vanderbilt UniversityNational Heart, Lung, and Blood Institute (NHLBI); University of South Florida and other collaboratorsCompletedHermansky Pudlak SyndromeUnited States
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Jesse RomanGenentech, Inc.UnknownInterstitial Lung Disease | Hermansky Pudlak SyndromeUnited States, Puerto Rico
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National Institute of Allergy and Infectious Diseases...WithdrawnColitis | Cytokines | Hermanski-Pudlak Syndrome | Lymphocytes | Drug EvaluationUnited States
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National Human Genome Research Institute (NHGRI)TerminatedPulmonary Fibrosis | Metabolic Disease | Oculocutaneous Albinism | Hermansky-Pudlak Syndrome (HPS) | Platelet Storage Pool DeficiencyUnited States
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Paul SzabolcsEnrolling by invitationChronic 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
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Mayo ClinicRecruitingCystinuria | Hyperoxaluria | Dent Disease | Lowe Syndrome | Adenine Phosphoribosyltransferase DeficiencyUnited States, Canada, Iceland, Israel
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New York Medical CollegeNational Institute of Neurological Disorders and Stroke (NINDS); National Institutes...Active, not recruitingChronic Fatigue Syndrome | Postural Tachycardia Syndrome (POTS) | Myalgic Encephalomyelitis | Systemic Exertion Intolerance Disease (SEID) | Neurally Mediated Syncope (NMS)United States
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SanofiPulse Infoframe Ltd.RecruitingNiemann-Pick Diseases | Acid Sphingomyelinase DeficiencyUnited States
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National Human Genome Research Institute (NHGRI)RecruitingPulmonary Fibrosis | Healthy Volunteers | Hermansky-Pudlak Syndrome (HPS)United States
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William Gahl, M.D.National Human Genome Research Institute (NHGRI)CompletedPulmonary Fibrosis | Inborn Errors of Metabolism | Albinism | Oculocutaneous Albinism | Platelet Storage Pool DeficiencyUnited States
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Albert Einstein College of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditionsUnited States
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National Institute of Allergy and Infectious Diseases...RecruitingChronic Granulomatous Disease (CGD) | X-Linked Severe Combined Immune Deficiency (XSCID) | Leukocyte Adhesion Deficiency 1 (LAD) | Graft Versus Host Disease (cGvHD)United States
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Weill Medical College of Cornell UniversityUnknownInterventional Endoscopy Database for Pancreatico-biliary, Gastrointestinal and Esophageal DisordersColorectal Cancer | Pancreatic Cancer | Esophageal Cancer | Cholangiocarcinoma | Bile Duct Cancer | Cholangitis | Barrett's Esophagus | Gallstones | Choledocholithiasis | Bile Leak | Obstructive Jaundice | Duodenal Cancer | Pancreatic Pseudocysts | Ampullary Cancer | Biliary Strictures | Recurrent Pancreatitis | Bile Duct... and other conditionsUnited States
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Richard FryeAutism Discovery and Treatment FoundationRecruitingEpilepsy | Neurodevelopmental Disorders | Autism Spectrum Disorder | Down Syndrome | Mitochondrial Encephalomyopathies | Pediatric Acute-Onset Neuropsychiatric Syndrome | Cerebral Folate Deficiency | Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal InfectionUnited States
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Fundación Española de Hematología y HemoterapíaRecruitingNiemann-Pick Diseases | Gaucher Disease | Splenomegaly | Acid SphingoMyelinase Deficiency | ASMDSpain
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Wake Forest University Health SciencesNational Heart, Lung, and Blood Institute (NHLBI)RecruitingKidney Diseases | Hypertension | Pediatric Obesity | Proteinuria | Albuminuria | Kidney Injury | Blood Pressure Disorders | Autonomic Dysfunction | Pediatric Kidney Disease | Left Ventricular Dysfunction | Left Ventricular Hypertrophy | Kidney Dysfunction | Autonomic Imbalance | Left Ventricular Diastolic Dysfunction and other conditionsUnited States
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Genzyme, a Sanofi CompanyTerminatedNiemann-Pick Disease | Acid Sphingomyelinase DeficiencyUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingCommon Variable Immunodeficiency | Chronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | X-linked Lymphoproliferative Disease | Langerhan's Cell Histiocytosis | Chediak-Higashi Syndrome | Griscelli Syndrome | Bare Lymphocyte Syndrome | SCID | Omenn's Syndrome | Reticular... and other conditionsUnited States
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Rigshospitalet, DenmarkUniversity College, LondonEnrolling by invitationEnd Stage Liver Disease | Cardiovascular Diseases | Fractures, Bone | Diabetes Mellitus | Renal Insufficiency | Hepatitis B | Hepatitis C | Kidney Failure, Chronic | Proteinuria | HIV | Coinfection | Acidosis, Lactic | AIDSSpain, Switzerland, Germany, United Kingdom, Austria, Sweden, Finland, Israel, Denmark, France, Italy, Iceland, Belgium, Slovakia, Norway, Lithuania, Portugal, Poland, Croatia, Czechia, Greece, Hungary, Luxembourg, Argentina, Belarus, Bosnia... and more
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Institut National de la Santé Et de la Recherche...RecruitingNeurofibromatosis Type 1 | Ichthyosis | Ectodermal Dysplasia | Albinism | Pemphigus | Mucous Membrane Pemphigoid | Inherited Epidermolysis Bullosa | Palmoplantar Keratoderma | Incontinentia PigmentiFrance
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Min-Sheng General HospitalCompletedLiver Diseases | Heart Failure | Stroke | Pneumonia | Septic Shock | Respiratory Failure | Trauma | Acute Kidney Injury | Diabetic Ketoacidosis | Head Injury | GastroIntestinal Bleeding | Intracerebral Hemorrhage | Heart Disease, IschemicTaiwan
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National Human Genome Research Institute (NHGRI)CompletedCancer | Myelofibrosis | Pulmonary Fibrosis | Gaucher Disease | Hermansky-Pudlak Syndrome (HPS)United States
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Region SkaneNot yet recruitingPneumonia | Burns | Shock | Poisoning | Abdominal Pain | Chest Pain | Trauma | Fever | Bradycardia | Syncope | Seizures | Vertigo | Tachycardia | Cardiac Arrest | Diabetic Ketoacidosis | Shortness of Breath | Allergic Reaction | Asthma Acute | Altered Mental Status | COPD Exacerbation Acute | Pulmonary Edema - Acute | Hyperglycemic Hyperosmolar...
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Khondrion BVJulius Clinical; ProPharma Group; CertaraCompletedMitochondrial Diseases | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Maternally Inherited Diabetes and Deafness (MIDD) | Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke Like Episodes (MELAS) | Chronic Progressive External Ophthalmoplegia (CPEO)Netherlands, Denmark, United Kingdom, Germany
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Maastricht University Medical CenterEnrolling by invitationTuberous Sclerosis | Basal Cell Nevus Syndrome | Cutis Laxa | Epidermolysis Bullosa | Ichthyosis | Ectodermal Dysplasia | Albinism | Xeroderma Pigmentosum | Birt-Hogg-Dube Syndrome | Palmoplantar KeratosesNetherlands