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Clinical Trials on Aromatic Amino Acid Decarboxylase Deficiency
Total 556 results
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Fondazione SISA (Societa Italiana per lo Studio...Recruiting
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Barcelona Institute for Global HealthCompletedAttention Deficit Hyperactivity Disorder | Child Behavior Problem | Fatty Acid Deficiency
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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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Promethera TherapeuticsCompletedUrea Cycle Disorders | Crigler Najjar SyndromeIsrael, United Kingdom, Belgium, France, Italy
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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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Shenzhen Geno-Immune Medical InstituteUnknownAdenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)China
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University of UtahNational Institute of Allergy and Infectious Diseases (NIAID); Duke University and other collaboratorsCompleted
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary 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 conditionsUnited States
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Children's Hospital Medical Center, CincinnatiNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedWolman Disease | Cholesterol Ester Storage Disease | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2United 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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National Human Genome Research Institute (NHGRI)Active, not recruitingWiskott- Aldrich Syndrome | ADA Deficient SCIDUnited States
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Assistance Publique - Hôpitaux de ParisRecruitingPediatric Patients | Any Type of Severe Combined Immunodeficiency (SCID) | Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)France
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National Human Genome Research Institute (NHGRI)Completed
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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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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD)United States, United Kingdom, France, Canada, Czech Republic, Italy, Poland, Switzerland
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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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Cellaion SATerminatedUrea Cycle Disorder | Crigler-Najjar Syndrome | Acute on Chronic Liver FailureBelgium, Spain, France, Bulgaria, Poland
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National Human Genome Research Institute (NHGRI)Withdrawn
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Assistance Publique - Hôpitaux de ParisCompletedOculocerebrorenal SyndromeFrance
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Chung-Hsing WangSanofiNot yet recruiting
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SanofiRecruitingAcid Sphingomyelinase Deficiency (ASMD)France
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Shenzhen Geno-Immune Medical InstituteUnknownSCID, X LinkedChina
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Genzyme, a Sanofi CompanyCompletedHuman Acid Sphingomyelinase DeficiencyUnited Kingdom, United States
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University Of PerugiaRecruitingObesity | Ketosis | Lysosomal Acid Lipase Deficiency | Ketogenic DietingItaly
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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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SanofiRecruitingAcid SphingoMyelinase Deficiency | Gaucher Disease, SplenomegalyFrance
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Genzyme, a Sanofi CompanyCompletedPompe Disease | Glycogen Storage Disease Type II (GSD II) | Acid Maltase DeficiencyUnited States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase DeficiencyUnited States, Canada, Australia
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Genzyme, a Sanofi CompanyTerminatedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase DeficiencyUnited States, Israel
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SanofiPulse Infoframe Ltd.RecruitingNiemann-Pick Diseases | Acid Sphingomyelinase DeficiencyUnited States
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Genzyme, a Sanofi CompanyTerminatedGlycogenosis 2 | Glycogen Storage Disease Type II (GSD II) | Acid Maltase Deficiency | Pompe Disease (Infantile-Onset)United States, Taiwan, Germany
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Genzyme, a Sanofi CompanyCompletedGlycogenesis 2 Acid Maltase Deficiency | Pompe Disease (Late-Onset) | Glycogen Storage Disease Type II (GSD II)United States, Netherlands, United Kingdom, Germany
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Genzyme, a Sanofi CompanyCompletedGlycogenesis 2 Acid Maltase Deficiency | Pompe Disease (Late-Onset) | Glycogen Storage Disease Type II (GSD II)France
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease (Late-Onset) | Glycogenesis Type II | Acid Maltase Deficiency (AMD)United States, France, Canada, Netherlands, Australia
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Spark TherapeuticsActive, not recruitingLysosomal Storage Diseases | Glycogen Storage Disease Type II | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase DeficiencyUnited States, Canada, Netherlands, France, Denmark, Germany, Italy, United Kingdom
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Spark TherapeuticsCompletedLysosomal Storage Diseases | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase DeficiencyUnited States, Italy, United Kingdom, Netherlands, France, Germany
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Genzyme, a Sanofi CompanyRecruitingPompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Glycogenesis 2 Acid Maltase DeficiencyCroatia, United States, Italy, Belgium, Czechia
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National Human Genome Research Institute (NHGRI)National Institute of Allergy and Infectious Diseases (NIAID); National Institute...CompletedCGD | Wiskott-Aldrich Syndrome | Primary Immunodeficiencies | APECED | SCIDUnited States
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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
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RWJ Barnabas Health at Jersey City Medical CenterUnknownLynch Syndrome | Endometrial Cancer | Somatic Mutation | Cancer Gene MutationUnited States
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Fundación Española de Hematología y HemoterapíaRecruitingNiemann-Pick Diseases | Gaucher Disease | Splenomegaly | Acid SphingoMyelinase Deficiency | ASMDSpain
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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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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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Genzyme, a Sanofi CompanyTerminatedNiemann-Pick Disease | Acid Sphingomyelinase 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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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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Maastricht University Medical CenterRecruitingQuality of Life | Tuberous Sclerosis | Basal Cell Nevus Syndrome | Cutis Laxa | Epidermolysis Bullosa | Ichthyosis | Ectodermal Dysplasia | Albinism | Birt-Hogg-Dube Syndrome | Palmoplantar KeratosesNetherlands
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University Hospital, BordeauxRecruitingIntellectual Disability | Cystic Fibrosis | Neurodegeneration With Brain Iron Accumulation (NBIA) | Congenital Heart Defect | Albinism | Rubinstein-Taybi Syndrome | Periventricular Nodular HeterotopiaFrance