Search clinical trials for: Orphan Diseases
Total 371991 results
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University Hospital, BonnSwedish Orphan BiovitrumRecruitingHaemophilia A | Synovitis | Hemophilia Arthropathy | SonographyGermany
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AOP Orphan Pharmaceuticals AGNot yet recruitingPulmonary Arterial HypertensionSpain, Austria, France, Hungary
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AOP Orphan Pharmaceuticals AGGCP-Service International Ltd. & Co. KG; ANOVA CRO s.r.o.; PharmaLex Belgium; Aixial...RecruitingPulmonary Arterial HypertensionAustria, Spain, France, Germany, Italy, Czechia, Hungary, Poland, Portugal, Romania
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Swedish Orphan BiovitrumRecruitingHereditary Tyrosinemia, Type IChina
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Imagine InstituteNot yet recruitingDysmorphia | Orphan Diseases
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University of LausanneRecruiting
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Swedish Orphan BiovitrumSyneos HealthRecruitingHemophilia A, SevereItaly, Ireland, Croatia, Greece, Spain, Sweden, Slovenia, United Kingdom, France, Germany, Norway, Austria, Czechia, Netherlands, Belgium
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Swedish Orphan BiovitrumCMIC Co, Ltd. JapanRecruitingStill's Disease, Adult-Onset | Still's Disease, Juvenile OnsetJapan
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Columbia UniversityFDA Office of Orphan Products DevelopmentRecruiting
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Swedish Orphan BiovitrumRecruitingParoxysmal Nocturnal HemoglobinuriaSpain
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Hanny Al-Samkari, MDSwedish Orphan BiovitrumRecruitingGastrointestinal Neoplasms | Gastrointestinal Cancer | Chemotherapy-Induced ThrombocytopeniaUnited States
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TVAX BiomedicalFDA Office of Orphan Products DevelopmentRecruitingGlioblastoma Multiforme of BrainUnited States
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University of BernUniversity of Zurich; Kantonsspital Aarau; Ente Ospedaliero Cantonale, Bellinzona and other collaboratorsRecruiting
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Jena University HospitalNovartis; Amgen; Grifols Biologicals, LLC; argenx; Swedish Orphan Biovitrum; University...Recruiting
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Swedish Orphan BiovitrumApellis Pharmaceuticals, Inc.RecruitingTransplant-Associated Thrombotic MicroangiopathyUnited States, France, Greece, Italy, Spain
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Second Hospital of Shanxi Medical UniversityNot yet recruiting
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Swedish Orphan BiovitrumRecruitingSystemic Lupus Erythematosus | Macrophage Activation Syndrome | Still Disease | Secondary Hemophagocytic Lymphohistiocytosis | SJIA | AOSD | MASUnited States, Belgium, Italy, Netherlands, United Kingdom, China, France, Spain, Czechia, Canada, Sweden, Japan, Germany, Poland
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Assistance Publique - Hôpitaux de ParisSwedish Orphan BiovitrumRecruiting
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University Hospital, BonnSwedish Orphan BiovitrumRecruiting
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University of PennsylvaniaInternational Foundation for CDKL5 Research; Loulou Foundation; CDKL5 AllianceRecruiting
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TaiwanJ Pharmaceuticals Co., LtdNot yet recruiting
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Fred Hutchinson Cancer CenterSwedish Orphan BiovitrumRecruitingB-Cell Non-Hodgkin LymphomaUnited States
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Stephan Grupp MD PhDUniversity of PennsylvaniaRecruitingPediatric and Young Adult Patientswith Hypodiploid or t(17;19) B-ALL | Infants With Very High Risk KMT2A B-ALL | Patients With Central Nervous System Relapse Who Did Not Receive Cranial Radiation or Bone Marrow TransplantationUnited States
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Northwell HealthSwedish Orphan BiovitrumRecruitingMeniere's Disease | Autoimmune Inner Ear DiseaseUnited States
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National Heart, Lung, and Blood Institute (NHLBI)RecruitingGenetic Predisposition | Vascular Dysfunction | Genetic MutationsUnited States
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Nicholas Ah MewIcahn School of Medicine at Mount Sinai; Boston Children's HospitalRecruitingN-acetylglutamate Synthase (NAGS) DeficiencyUnited States
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Boston Children's HospitalRecruitingRare Disorders | Orphan Diseases | Undiagnosed ConditionsUnited States
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Kevin KilgoreNational Institute of Neurological Disorders and Stroke (NINDS); Case Western... and other collaboratorsRecruitingSpinal Cord Injuries | Spinal Cord Injury at C5-C7 Level | Spinal Cord Injury Cervical | Spinal Cord Injury at C5-C7 Level With Complete Lesion | Spinal Cord Injury at C5-C7 Level With Incomplete LesionUnited States
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial 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 conditionsUnited States, Australia
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Vicore Pharma ABOrphan ReachCompletedIdiopathic Pulmonary FibrosisUnited Kingdom, India, Ukraine, Russian Federation
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Swedish Orphan BiovitrumActive, not recruitingHemophilia ASpain, Germany, Czechia, Italy, Estonia, Finland, Greece, Netherlands, Saudi Arabia, Slovenia, Sweden, Switzerland, United Kingdom
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Swedish Orphan BiovitrumSuspendedCold Agglutinin DiseaseSpain, Belgium, Norway, United States, Japan, United Kingdom, Netherlands, Canada, Hungary, Austria, Finland, Georgia, Germany, Italy
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Swedish Orphan BiovitrumCompletedStudy to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic LymphohistiocytosisPrimary Hemophagocytic LymphohistiocytosisSpain, United States, United Kingdom, Germany, Italy, Sweden, Canada, Switzerland
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AlbireoCompletedPrimary Biliary Cirrhosis | Alagille Syndrome | Progressive Familial Intrahepatic Cholestasis | Orphan Cholestatic Liver DiseasesUnited Kingdom
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Daiichi Sankyo Co., Ltd.Orphan Disease Treatment Institute Co., Ltd.Completed
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AlbireoCompletedPrimary Biliary Cirrhosis | Alagille Syndrome | Progressive Familial Intrahepatic Cholestasis | Orphan Cholestatic Liver Diseases
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Swedish Orphan BiovitrumCompletedHaemophilia A | Haemophilia BSweden, Finland, Denmark
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Lundquist Institute for Biomedical Innovation at...Swedish Orphan Biovitrum; Cure Sanfilippo FoundationCompleted
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Swedish Orphan BiovitrumActive, not recruitingPrimary Hemophagocytic LymphohistiocytosisChina
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Swedish Orphan BiovitrumCompletedChronic GoutUnited States
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Swedish Orphan BiovitrumCompletedChronic GoutUnited States, Serbia, Georgia, Ukraine, Russian Federation
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Swedish Orphan BiovitrumActive, not recruitingImmune Thrombocytopenia | ITPCroatia, Czechia, Germany, Italy, Netherlands, Norway, Spain, Switzerland, United Kingdom
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Swedish Orphan BiovitrumCerner EnvizaCompleted
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Swedish Orphan BiovitrumCerner EnvizaCompletedHemophilia A With InhibitorFrance, Ireland, Italy, Norway, Germany, Kuwait, Saudi Arabia, Switzerland
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Swedish Orphan BiovitrumCerner EnvizaActive, not recruitingHemophilia BCzechia, Greece, Ireland, Italy, Norway, Saudi Arabia, Spain, Sweden, United Kingdom
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Swedish Orphan BiovitrumCerner EnvizaCompleted
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Imagine InstituteCompletedOrphan Diseases | DysmorphologyFrance
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Memorial Sloan Kettering Cancer CenterSwedish Orphan BiovitrumActive, not recruitingLeukemia | Multiple Myeloma | Non-Hodgkin's Lymphoma | Myelodysplastic SyndromeUnited States
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Oncternal Therapeutics, IncUniversity of California, San Diego; Pharmacyclics LLC.; California Institute...Active, not recruitingMantle Cell Lymphoma | Marginal Zone Lymphoma | B-cell Chronic Lymphocytic Leukemia | Small Lymphocytic LymphomaUnited States
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Swedish Orphan BiovitrumIQVIA Pvt. LtdTerminatedHemophilia A | Hemophilia BBulgaria, Croatia, Czechia, France, Hungary, Ireland, Italy, Romania, Slovenia, Spain, United Kingdom