Search clinical trials for: Orphan Medical
Total 111471 results
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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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Second Hospital of Shanxi Medical UniversityRecruiting
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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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TVAX BiomedicalFDA Office of Orphan Products DevelopmentRecruitingGlioblastoma Multiforme of BrainUnited States
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Jena University HospitalNovartis; Amgen; Grifols Biologicals, LLC; argenx; Swedish Orphan Biovitrum; University...Recruiting
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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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Second Hospital of Shanxi Medical UniversityRecruitingCognitive DysfunctionChina
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Fred Hutchinson Cancer CenterSwedish Orphan BiovitrumRecruitingB-Cell Non-Hodgkin LymphomaUnited 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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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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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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Lundquist Institute for Biomedical Innovation at...Swedish Orphan Biovitrum; Cure Sanfilippo FoundationCompleted
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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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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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Wuerzburg University HospitalHannover Medical School; Goethe University; University Hospital Tuebingen; Universitätsklinikum... and other collaboratorsCompletedRare Diseases | Orphan DiseasesGermany
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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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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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MetroHealth Medical CenterVA Office of Research and Development; Case Western Reserve University; FDA Office...Active, not recruitingSpinal Cord Injury | TetraplegiaUnited States
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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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Philipps University Marburg Medical CenterDeutsche Krebshilfe e.V., Bonn (Germany); AOP Orphan Pharmaceuticals AGCompletedChronic Myeloid Leukemia in RemissionFrance, Germany
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The University of Texas Medical Branch, GalvestonCompletedPorphyria Cutanea TardaUnited States
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Stony Brook UniversityFDA Office of Orphan Products Development; State University of New YorkCompletedSepsis | Respiratory Distress Syndrome | Respiratory Distress Syndrome, AdultUnited States
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Second Hospital of Shanxi Medical UniversityCompleted
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Weill Medical College of Cornell UniversityFDA Office of Orphan Products Development; The Leukemia and Lymphoma Society; Karyopharm Therapeutics IncCompletedDiffuse Large B-Cell LymphomaUnited States
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Bioverativ, a Sanofi companySwedish Orphan BiovitrumCompletedHemophilia A With InhibitorsUnited States, Spain, Canada, Belgium, France, Bulgaria, Italy, United Kingdom, Japan, Germany
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Weill Medical College of Cornell UniversitySwedish Orphan BiovitrumWithdrawnCovid19 | Mechanical Ventilation Complication | Cytokine StormUnited States
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Shanghai Junshi Bioscience Co., Ltd.Iqvia Pty Ltd; TopAlliance Biosciences, Inc.Active, not recruitingAdvanced MalignanciesUnited States
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Swedish Orphan BiovitrumCompletedSanfilippo Syndrome Type A (MPS IIIA)United States, Germany, Turkey
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Swedish Orphan BiovitrumTerminatedStill's Disease, Adult-Onset | Still's Disease, Juvenile-OnsetUnited States, Canada
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Vicore Pharma ABOrphan ReachCompleted
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AOP Orphan Pharmaceuticals AGPharmaEssentia Corporation (for the U.S.)CompletedPolycythemia VeraSpain, Austria, Bulgaria, France, Germany, Hungary, Poland, Romania, Russian Federation, Slovakia, Ukraine, Czechia
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Martin Andreas, M.D.National Bank of Austria; Orphan MedicalCompleted
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Emmaus Medical, Inc.FDA Office of Orphan Products DevelopmentCompletedSickle Cell Anemia | ThalassemiaUnited States
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Bioverativ Therapeutics Inc.Swedish Orphan Biovitrum; Syntonix Pharmaceuticals, Inc.Completed
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PharmaxisCompletedCystic FibrosisUnited States, Spain, Israel, Belgium, Czechia, South Africa, Italy, Mexico, Argentina, Australia, Canada, Hungary, New Zealand, Poland, Romania, Russian Federation, Slovakia, Ukraine
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PTC TherapeuticsCystic Fibrosis FoundationCompletedCystic FibrosisUnited States, France, Belgium, Spain, Canada, Germany, Israel, Italy, Netherlands, Sweden, United Kingdom
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Swedish Orphan BiovitrumCompletedAcute Gouty ArthritisUnited States
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Second Hospital of Shanxi Medical UniversityCompletedIntraspinal AnesthesiaChina
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PTC TherapeuticsCystic Fibrosis FoundationCompletedCystic FibrosisUnited States, France, Belgium, United Kingdom, Spain, Canada, Germany, Israel, Italy, Netherlands, Sweden
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RedHill Biopharma LimitedMedical University of South Carolina; National Cancer Institute (NCI); FDA Office... and other collaboratorsCompletedPancreatic Cancer | Unspecified Adult Solid Tumor, Protocol SpecificUnited States
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Symphogen A/SSwedish Orphan BiovitrumCompletedImmune Thrombocytopenic PurpuraUnited States, Belgium, Germany, India, Israel, Poland, Romania, Russian Federation, Serbia, Spain, Ukraine, United Kingdom