Search clinical trials for: orphan drug
Total 258586 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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Second Hospital of Shanxi Medical UniversityRecruiting
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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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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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Mclean HospitalNational Institute of Mental Health (NIMH)RecruitingMajor Depressive DisorderUnited States
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Xenthera, Inc.Not yet recruitingAdvanced Solid Tumor | Metastatic Solid Tumor
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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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Assistance Publique - Hôpitaux de ParisSwedish Orphan BiovitrumRecruiting
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Second Hospital of Shanxi Medical UniversityRecruitingCognitive DysfunctionChina
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University Hospital, BonnSwedish Orphan BiovitrumRecruiting
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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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Nicholas Ah MewIcahn School of Medicine at Mount Sinai; Boston Children's HospitalRecruitingN-acetylglutamate Synthase (NAGS) DeficiencyUnited States
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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 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 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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Memorial Sloan Kettering Cancer CenterSwedish Orphan BiovitrumActive, not recruitingLeukemia | Multiple Myeloma | Non-Hodgkin's Lymphoma | Myelodysplastic SyndromeUnited States
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Swedish Orphan BiovitrumPRA Health Sciences; Cromsource; BioMérieux; Q2 Solutions; Cytel Inc.; ABF Pharmaceutical...Terminated
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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
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University of CincinnatiNational Center for Research Resources (NCRR); Office of Rare Diseases (ORD); FDA Office of Orphan Products DevelopmentCompletedLymphangioleiomyomatosisUnited States, Canada, Japan
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Swedish Orphan BiovitrumLight Chain Bioscience - Novimmune SATerminatedHemophagocytic LymphohistiocytosesUnited States
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Weill Medical College of Cornell UniversityChildren's Hospital Colorado; SLC6A1 Connect; STXBP1 Foundation; Clara Inspired; University... and other collaboratorsEnrolling by invitationDevelopmental and Epileptic Encephalopathy | STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental DisorderUnited States
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Melissa DelbelloPatient-Centered Outcomes Research InstituteCompletedBipolar DisorderUnited States
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Swedish Orphan BiovitrumHarvard Medical School (HMS and HSDM); Brigham and Women's HospitalCompletedHemophagocytic LymphohistiocytosesUnited States
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Swedish Orphan BiovitrumCompletedHealthy VolunteersUnited Kingdom
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Fondazione per la Ricerca Ospedale MaggioreAOP Orphan Pharmaceuticals AGCompleted
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Boston Children's HospitalFDA Office of Orphan Products DevelopmentActive, not recruiting
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Gradalis, Inc.TerminatedSarcoma | Neoplasms | Neoplasms, Connective and Soft Tissue | Neoplasms by Histologic Type | Sarcoma, Ewing | Neoplasms, Bone Tissue | Neoplasms, Connective Tissue | Ewing Sarcoma | Rare Diseases | Ewing's Tumor Metastatic | Ewing Family of Tumors | Ewing's Sarcoma Metastatic | Ewing's Tumor RecurrentUnited States
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Oncternal Therapeutics, IncPharmacyclics LLC.WithdrawnImmune System Diseases | Lymphoma | Lymphoma, B-Cell | Lymphoproliferative Disorders | Lymphatic Diseases | Immunoproliferative Disorders | Lymphoma, Non-Hodgkin | Lymphoma, Mantle-Cell
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The Cleveland ClinicActive, not recruitingLiver TransplantationUnited States
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Philipps University Marburg Medical CenterDeutsche Krebshilfe e.V., Bonn (Germany); AOP Orphan Pharmaceuticals AGCompletedChronic Myeloid Leukemia in RemissionFrance, Germany