- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials Sponsored by Orphan Therapeutics
Total 6614 results
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Boston Children's HospitalFDA Office of Orphan Products DevelopmentActive, not recruiting
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Aegerion Pharmaceuticals, Inc.FDA Office of Orphan Products DevelopmentCompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, South Africa
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University of ChicagoCompleted
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University of FloridaCompleted
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National Jewish HealthCompletedMycobacterium Avium-intracellulare Infection
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Johns Hopkins UniversityCompletedCerebral HemorrhageUnited States
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University of MichiganCompletedGraft Versus Host DiseaseUnited States
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University of TexasTerminated
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University of TexasCompletedAcute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Chronic Myelogenous Leukemia
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University of PittsburghCompletedTrigeminal NeuralgiaUnited States
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Holles Laboratories, Inc.Completed
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University of VermontCompleted
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University of TexasCompletedEsophageal Achalasia
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Akebia TherapeuticsCyclerion TherapeuticsCompletedType 2 Diabetes Mellitus With Diabetic NephropathyUnited States
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Akebia TherapeuticsCyclerion TherapeuticsCompletedHeart Failure With Preserved Ejection FractionUnited States, Canada
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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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Weill Medical College of Cornell UniversitySwedish Orphan BiovitrumWithdrawnCovid19 | Mechanical Ventilation Complication | Cytokine StormUnited States
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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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NeuroHealing Pharmaceuticals Inc.FDA Office of Orphan Products DevelopmentSuspended
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AOP Orphan Pharmaceuticals AGPharmaEssentia (Co-Sponsor for USA)CompletedPolycythemia VeraSpain, Belgium, Austria, Italy, Bulgaria, Czech Republic, France, Germany, Hungary, Poland, Romania, Russian Federation, Slovakia, Ukraine
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University of PittsburghCompletedSezary Syndrome | Cutaneous T-cell LymphomaUnited States
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FDA Office of Orphan Products DevelopmentUniversity of UtahCompleted
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University of ChicagoCompleted
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University of IowaCompletedLymphatic MalformationsUnited States
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University of TexasCompleted
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The Cleveland ClinicCompleted
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FDA Office of Orphan Products DevelopmentBoston Children's HospitalCompleted
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University of TexasCompletedAcute Renal FailureUnited States
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University of PittsburghCompletedMycosis Fungoides | Sezary Syndrome | Cutaneous T-cell LymphomaUnited States
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University of WashingtonCompleted
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Johns Hopkins UniversityCompletedEpilepsy | Cysticercosis
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Bellus Health IncFDA Office of Orphan Products DevelopmentCompletedGastrointestinal Diseases | Kidney Diseases | Rheumatoid Arthritis | Nephrotic Syndrome | Secondary (AA) Amyloidosis | Familial Mediterranean SyndromeUnited States, Israel, Finland, France, Italy, Lithuania, Netherlands, Poland, Russian Federation, Spain, Turkey, United Kingdom
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Boston Children's HospitalFDA Office of Orphan Products DevelopmentTerminated
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Swedish Orphan BiovitrumPRA Health Sciences; BioMérieux; Cytel Inc.TerminatedGraft FailureItaly, Belgium, United Kingdom, France, Germany, Netherlands
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Emmaus Medical, Inc.FDA Office of Orphan Products DevelopmentCompletedSickle Cell Anemia | ThalassemiaUnited States
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IWK Health CentreCompletedPertussis | Whooping Cough
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Ohio State UniversityCompletedMuscular Dystrophy, Facioscapulohumeral
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University of IowaCompleted
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FDA Office of Orphan Products DevelopmentLeadiant Biosciences, Inc.Completed
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Boston Children's HospitalCompletedSickle Cell AnemiaUnited States
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The Cleveland ClinicTerminated
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University of FloridaCompleted
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University of MichiganCompleted
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Seattle Children's HospitalCompletedPain | Infant, Newborn, Diseases
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Massachusetts General HospitalCompleted
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University of PennsylvaniaTerminatedOrnithine Transcarbamylase Deficiency Disease
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University of TennesseeFDA Office of Orphan Products DevelopmentCompleted
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University of ArizonaFDA Office of Orphan Products DevelopmentTerminated
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Eikon TherapeuticsImpact Therapeutics, Inc.RecruitingAdvanced Solid TumorAustralia, United States
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APRINOIA Therapeutics, LLCActive, not recruitingAlzheimer Disease | Tauopathies | Healthy VolunteersUnited States