Clinical Trials on Inborn Error of Bile Acid Metabolism
Total 3066 results
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Hugo W. Moser Research Institute at Kennedy Krieger...TerminatedAdrenoleukodystrophyUnited States
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Onofre, Aurora Pujol, M.D.Instituto de Salud Carlos III; Fundacion Hesperia; ELA España AssociationCompletedAdrenomyeloneuropathy | X-linked AdrenoleukodystrophySpain
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Onofre, Aurora Pujol, M.D.Ministerio de Sanidad, Servicios Sociales e Igualdad; Fundacion HesperiaCompleted
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Hugo W. Moser Research Institute at Kennedy Krieger...European Leukodystrophy AssociationCompletedX-linked AdrenoleukodystrophyUnited States
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National Human Genome Research Institute (NHGRI)Withdrawn
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University Hospital OlomoucUniversity Hospital Ostrava; Tomas Bata Hospital, Czech Republic; General University... and other collaboratorsRecruitingCushing Syndrome | Pheochromocytoma | Adrenocortical Carcinoma | Adrenal Cortex Diseases | Adrenocortical Adenoma | Adrenal Tumor | Adrenalectomy; Status | Adrenal Mass | Adrenal Hyperplasia | Adrenal Disease | Adrenocortical Tumor | Conn Adenoma | Adrenal Gland MetastasisCzechia
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Alice BertainaCalifornia Institute for Regenerative Medicine (CIRM)RecruitingCKD Stage 4 | Cystinosis | FSGS | SLE Nephritis | SIODUnited States
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AstraZenecaRecruitingTransthyretin Amyloidosis Cardiomyopathy, Heart Failure With Preserved Ejection FractionRussian Federation
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Alnylam PharmaceuticalsCompletedATTR Amyloidosis | Transthyretin (TTR)-Mediated Amyloidosis | Familial Amyloidotic Polyneuropathy (FAP) | Familial Amyloid NeuropathiesFrance, Germany, Portugal, United Kingdom, Sweden, Spain
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National Institute of Neurological Disorders and...CompletedHealthy | Motor Neuron Disease | Lysosomal Storage Disease | Movement Disorder | Demyelinating DiseaseUnited States
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Mayo ClinicCompleted
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CENTOGENE GmbH RostockWithdrawnTransthyretin Amyloidosis | Transthyretin Amyloid Cardiopathy | Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyIndia, Germany
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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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Assiut UniversityNot yet recruitingOxidative Stress in Patient With Haemoglobinopathy and Affect it of Endocrine System
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National Medical Research Center for Therapy and...Moscow State University of Medicine and DentistryRecruitingMedication Adherence | Adherence, Medication | Treatment Adherence | Familial Hypercholesterolemia | Motivational Interviewing | Adherence, Patient | Treatment Adherence and Compliance | Patient Compliance | Adherence | Hypercholesterolemia, Familial | Patient Adherence | Hypercholesterolemia, Autosomal Dominant and other conditionsRussian Federation
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Talaris Therapeutics Inc.Duke UniversityTerminatedNiemann-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
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Mirum Pharmaceuticals, Inc.Children's Hospital Medical Center, CincinnatiCompletedCholestasis | Peroxisomal Disorders | Adrenoleukodystrophy | Zellweger Syndrome | Infantile Refsum's DiseaseUnited States
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CENTOGENE GmbH RostockWithdrawnLung Disease | Cerebral Aneurysm | Macroglossia | Cardiac Diseases | Obstructive Sleep Apnoea | Muscular Weakness | Hepato-splenomegalyGermany, India, Sri Lanka
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Guy's and St Thomas' NHS Foundation TrustKing's College LondonNot yet recruitingDiabetes Mellitus | Intrahepatic Cholestasis of Pregnancy | Metabolic Disease
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SwanBio Therapeutics, Inc.RecruitingAMN | AMN Gene Mutation | X-ALDNetherlands, United States
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bluebird bioActive, not recruitingCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
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Centre Hospitalier Universitaire de NīmesCompletedCerebral Amyloid Angiopathy | SiderosisFrance
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Neurogene Inc.TerminatedCharcot-Marie-Tooth Disease | Hereditary Motor and Sensory Neuropathy IVUnited States
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NeuroVia, Inc.UnknownX-Linked AdrenoleukodystrophyUnited States
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Groupe Hospitalier Paris Saint JosephCompleted
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LMU KlinikumCompletedHealthy | Myotonic Dystrophy Type 2 | Pompe Disease (Late-onset) | Spinal Muscular Atrophy Type 3 | Myotonic Dystrophy Type 1 (DM1) | Facioscapulohumeral Muscular Dystrophy 1 | Inclusion Body Myositis, SporadicGermany
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bluebird bioTerminatedCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, United Kingdom, Argentina, Canada, Germany, Italy, Netherlands
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National Taiwan University HospitalCompletedAlzheimer Disease | Intracranial Hemorrhages | Cerebral Amyloid AngiopathyTaiwan
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National Center for Research Resources (NCRR)University of TexasCompleted
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Indiana Kidney Stone InstituteUniversity of Chicago; Indiana University School of MedicineRecruitingHyperparathyroidism | Cystinuria | Renal Calculi | Hypercalciuria | NephrocalcinosisUnited States
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PfizerCompletedHereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed PhenotypeUnited States
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Walter Reed Army Medical CenterUnknownLevator Ani Syndrome With Pain and/or Constipation Symptoms.United States
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Minoryx Therapeutics, S.L.RecruitingX-linked AdrenoleukodystrophyFrance
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Massachusetts General HospitalEuropean Leukodystrophy AssociationRecruitingRestless Legs Syndrome | AdrenoleukodystrophyUnited States, Netherlands
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National Taiwan University HospitalRecruitingAlzheimer Disease | Intracranial Hemorrhages | Cerebral Amyloid AngiopathyTaiwan
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Minoryx Therapeutics, S.L.Active, not recruitingCerebral AdrenoleukodystrophySpain, Germany, France, Argentina
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Thomas S. ScanlanWithdrawnAdrenomyeloneuropathy | X-Linked AdrenoleukodystrophyUnited States
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Assistance Publique - Hôpitaux de ParisCompleted
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Jasper Therapeutics, Inc.Recruiting
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Axcella Health, IncCompletedSickle Cell Disease | Anemia, Sickle Cell | Sickle Cell Anemia | To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease | Sickle Cell DisordersUnited States
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PfizerCompletedTransthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) | Transthyretin Cardiomyopathy (TTR-CM) | Familial Amyloid Cardiomyopathy | Senile Systemic Amyloidosis (SSA)
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Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | Amyloidosis, Hereditary | Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | Amyloidosis, Hereditary, Transthyretin-Related | Familial Transthyretin Cardiac AmyloidosisUnited States, France, Italy, Spain, Canada, Germany, Belgium, Sweden, United Kingdom
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National Taiwan University HospitalNational Cheng-Kung University HospitalUnknownMELAS Syndrome | Noninsulin-dependent Diabetes Mellitus With DeafnessTaiwan
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Assistance Publique - Hôpitaux de ParisCompleted
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Protecting Brains Saving FuturesNot yet recruitingBrain Injuries | Intraventricular Hemorrhage | Congenital Heart Disease | Hypoxic-Ischemic Encephalopathy | Newborn Morbidity | Inborn Errors of Metabolism | Extracorporeal Membrane Oxygenation Complication | Respiratory Complication | Hemodynamic Instability | Nosocomial Infection | Neonatal Seizure | Brain... and other conditionsBrazil
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National Taiwan University HospitalUnknown
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Assistance Publique - Hôpitaux de ParisNot yet recruiting
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CENTOGENE GmbH RostockAlnylam PharmaceuticalsRecruitingCardiomyopathies | Polyneuropathies | Transthyretin Amyloidosis | Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyGermany, Austria, Switzerland