Clinical Trials on Familial Hemophagocytic Lymphohistiocytosis
Total 1238 results
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Intellia TherapeuticsRecruitingTransthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac AmyloidosisUnited Kingdom, New Zealand, Sweden
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Intellia TherapeuticsActive, not recruitingTransthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac AmyloidosisFrance, United Kingdom, New Zealand, Sweden
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Novartis PharmaceuticalsCompletedElevated Cholesterol | Homozygous Familial Hypercholesterolemia | Heterozygous Familial Hypercholesterolemia | ASCVDUnited States, Canada, Czechia, Denmark, Germany, Hungary, Netherlands, Poland, South Africa, Spain, Sweden, Ukraine, United Kingdom
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REGENXBIO Inc.National Heart, Lung, and Blood Institute (NHLBI)TerminatedHomozygous Familial Hypercholesterolemia (HoFH)United States, Canada, Italy, Netherlands
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Emory UniversityCompletedSickle Cell Disease | Fanconi Anemia | Hurler Syndrome | Chronic Granulomatous Disease | Severe Congenital Neutropenia | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Thalassemia Major | Leukocyte... and other conditionsUnited States
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Seoul National University HospitalRecruitingFamilial Exudative VitreoretinopathiesKorea, Republic of
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Mirum Pharmaceuticals, Inc.Active, not recruitingProgressive Familial Intrahepatic Cholestasis (PFIC)United States, Argentina, France, Singapore, United Kingdom, Belgium, Turkey, Austria, Brazil, Canada, Colombia, Germany, Italy, Lebanon, Mexico, Poland
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Mirum Pharmaceuticals, Inc.CompletedProgressive Familial Intrahepatic Cholestasis (PFIC)Italy, United States, Argentina, France, Singapore, United Kingdom, Austria, Brazil, Mexico, Lebanon, Germany, Turkey, Poland, Belgium, Canada, Colombia, Hungary
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TakedaActive, not recruitingProgressive Familial Intrahepatic Cholestasis (PFIC)Japan
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Mirum Pharmaceuticals, Inc.WithdrawnProgressive Familial Intrahepatic Cholestasis (PFIC)
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CENTOGENE GmbH RostockWithdrawnTransthyretin Amyloidosis | Transthyretin Amyloid Cardiopathy | Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyIndia, Germany
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University of British ColumbiaVancouver Coastal Health Research Institute; Genome British ColumbiaRecruitingAcute Coronary Syndrome | Familial Hypercholesterolemia | STEMI | NSTEMI - Non-ST Segment Elevation MI | Familial Hypercholesterolemia - Heterozygous | Familial Hypercholesterolemia Due to Genetic Defect of Apolipoprotein B | Familial Hypercholesterolemia Due to Heterozygous LDL Receptor Mutation and other conditionsCanada
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Washington University School of MedicineIncyte CorporationNo longer available
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Instituto Aragones de Ciencias de la SaludHospital Clinic of Barcelona; Instituto de Salud Carlos IIICompletedObesity | Weight Loss | Familial Hypercholesterolemias | Familial Combined Hypercholesterolemia
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PfizerCompletedFamilial Amyloid PolyneuropathyUnited Kingdom, Spain, United States, Argentina, Brazil, France, Germany, Portugal, Sweden
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Ionis Pharmaceuticals, Inc.CompletedAmyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | TransthyretinUnited States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
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Ionis Pharmaceuticals, Inc.CompletedAmyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | TransthyretinNew Zealand, United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
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Novartis PharmaceuticalsTerminatedFamilial Chylomicronemia Syndrome (FCS) (HLP Type I)South Africa, Germany, United Kingdom, France, United States, Canada, Netherlands
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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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Centre Hospitalier Universitaire de NiceWithdrawnFamilial AmyloidosisFrance
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Novartis PharmaceuticalsCompletedFamilial Chylomicronemia Syndrome (FCS)South Africa, Spain, France, Germany, United Kingdom, United States, Canada, Netherlands
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Capital Medical UniversityPeking University First Hospital; The First Affiliated Hospital of Anhui Medical... and other collaboratorsRecruitingAlzheimer Disease | Familial Alzheimer Disease (FAD)China
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Assistance Publique Hopitaux De MarseilleCompletedAmyloid Neuropathies | Transthyretin AmyloidosisFrance
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Nantes University HospitalPfizerCompletedTransthyretin Amyloidosis | Familial Amyloid NeuropathyFrance
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University of Illinois at ChicagoWithdrawnVHL Gene Mutation | Polycythemia; Familial | Erythrocytosis; Familial | HIF-2alpha Erythrocytosis | PHD2 Erythrocytosis | Chuvash Erythrocytosis
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University Hospital, Basel, SwitzerlandCompletedMetabolic Syndrome | Familial Combined Hyperlipidemia | Familial HypertriglyceridemiaSwitzerland
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University Hospital, MontpellierCompleted
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PfizerCompletedFamilial Amyloid Polyneuropathy | ATTR-PNSweden, Brazil, Germany, Argentina, France, Portugal
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Kyoto UniversityOsaka University; Mie University; Tokushima University; Tokyo Metropolitan Geriatric... and other collaboratorsCompletedFamilial Alzheimer Disease (FAD) | PSEN1 MutationJapan
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AmgenCompleted
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Institute of Health Information and Statistics...Centre of Cardiovascular and Transplantation Surgery, Czech Republic; The Central... and other collaboratorsRecruitingFamilial HypercholesterolaemiaCzechia
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Vanderbilt University Medical CenterRecruitingIdiopathic Pulmonary Fibrosis | Familial Pulmonary Fibrosis | Familial Interstitial PneumoniaUnited States
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Fundación Hipercolesterolemia FamiliarInstituto de Salud Carlos III; Fundación Centro Nacional de Investigaciones...RecruitingFamilial HypercholesterolaemiaSpain
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Kastle Therapeutics, LLCCompletedHypercholesterolemia | Heterozygous FamilialUnited States, Italy, Poland, Ukraine, United Kingdom, Germany, Hong Kong, Taiwan, Spain, Argentina, Belgium, Israel, Malaysia, South Africa, Turkey, Netherlands, Korea, Republic of, Canada, New Zealand, Russian Federation, Brazil, Austral... and more
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedHypercholesterolemia, FamilialUnited States, Netherlands
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Novartis PharmaceuticalsRecruitingHypercholesterolaemia | Familial HypercholesterolaemiaJapan
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AmgenCompletedFamilial HypercholesterolemiaCanada, Italy, Norway, Belgium, United States, Turkey, Australia, Austria, South Africa, Poland, Switzerland, Spain, Portugal, Slovenia, Brazil, Russian Federation, Hungary, United Kingdom, Colombia, Czechia, Netherlands, Greece, Ma...
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa, Australia, Austria, Canada, Czechia, France, Greece, Italy, Japan, Ukraine
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Shanghai Junshi Bioscience Co., Ltd.CompletedHeterozygous Familial HypercholesterolemiaChina
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Mirum Pharmaceuticals, Inc.CompletedProgressive Familial Intrahepatic Cholestasis (PFIC)United States, United Kingdom, Poland, France
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Daniel GaudetUltragenyx Pharmaceutical IncActive, not recruitingSafety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial HypercholesterolemiaHomozygous Familial HypercholesterolemiaCanada
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Innovent Biologics (Suzhou) Co. Ltd.Completed
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Amryt PharmaActive, not recruitingHomozygous Familial Hypercholesterolaemia (HoFH)Germany, Israel, Italy, Saudi Arabia, Spain, Tunisia
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AkesoAD Pharmaceuticals Co., Ltd.CompletedHeterozygous Familial HypercholesterolemiaChina
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Nantes University HospitalCompletedFamilial HypobetalipoproteinemiaFrance
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Nantes University HospitalCompletedStudy of cardiovAscular Contrasted Phenotypes in Patients With FamIliaI hypercholesteRolemia (SAFIR)Homozygous Familial HypercholesterolemiaFrance
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The Medicines CompanyCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, Canada
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Madrigal Pharmaceuticals, Inc.CompletedHeterozygous Familial HypercholesterolemiaDenmark