Clinical Trials Sponsored by Porphyria Rare Disease Clinical Research Consortium
Total 92 results
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University of Alabama at BirminghamPorphyria Rare Disease Clinical Research ConsortiumCompletedErythropoietic Protoporphyria (EPP)United States
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Office of Rare Diseases (ORD)Rare Diseases Clinical Research NetworkUnknownMyelodysplastic Syndromes | Purpura, Thrombocytopenic | Anemia, Aplastic | Red-Cell Aplasia, Pure | Bone Marrow Failure Syndromes | Hemoglobinuria, Paroxysmal | Leukemia, LymphocyticUnited States
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Office of Rare Diseases (ORD)Rare Diseases Clinical Research NetworkUnknown
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Office of Rare Diseases (ORD)Rare Diseases Clinical Research NetworkUnknownMyelodysplastic SyndromeUnited States
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National Institute of Allergy and Infectious Diseases...Rare Diseases Clinical Research Network; Primary Immune Deficiency Treatment...Enrolling by invitationGranulomatous Disease, ChronicUnited States, Canada
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National Institute of Allergy and Infectious Diseases...Rare Diseases Clinical Research Network; Primary Immune Deficiency Treatment...CompletedWiskott-Aldrich SyndromeUnited States, Canada
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National Institute of Allergy and Infectious Diseases...Infectious Diseases Clinical Research ConsortiumRecruiting
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University of California, Los AngelesRare Diseases Clinical Research Network; Office of Rare Diseases (ORD)CompletedCerebellar Diseases | Episodic Ataxia SyndromeUnited States, Canada, United Kingdom
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National Institute of Arthritis and Musculoskeletal...The Cleveland Clinic; Rare Diseases Clinical Research Network; Office of Rare...CompletedGiant Cell Arteritis | Takayasu's ArteritisUnited States, Canada
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University of RochesterNational Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaboratorsCompletedAndersen-Tawil Syndrome | Andersen SyndromeUnited States, Canada, Italy, United Kingdom
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Andrea GropmanNational Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaboratorsCompletedBrain Diseases, Metabolic, Inborn | Ornithine Transcarbamylase Deficiency | Urea Cycle DisorderUnited States
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Baylor College of MedicineRare Diseases Clinical Research Network; Neogenis LaboratoriesCompletedUrea Cycle Disorders, Inborn | Urea Cycle Disorder | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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Richard Barohn, MDRare Diseases Clinical Research Network; Office of Rare Diseases (ORD)CompletedMyotonic Disorders | Myotonia Congenita | Nondystrophic MyotoniasCanada, United States, United Kingdom
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Phillip LowNational Institute of Neurological Disorders and Stroke (NINDS); Vanderbilt... and other collaboratorsTerminated
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University of PennsylvaniaNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaboratorsRecruitingGiant Cell Arteritis | Microscopic Polyangiitis | Polyarteritis Nodosa | Takayasu's Arteritis | Eosinophilic Granulomatosis With Polyangiitis (Churg-Strauss) | Granulomatosis With Polyangiitis (Wegener's)United States, Canada, Turkey
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University of PennsylvaniaNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaboratorsCompletedWegener's GranulomatosisUnited States
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University of PennsylvaniaNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaboratorsCompletedTemporal ArteritisUnited States, Canada
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University of PennsylvaniaNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaboratorsCompletedPolyarteritis NodosaUnited States, Canada
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University of PennsylvaniaNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaboratorsCompletedMicroscopic Polyangiitis | Granulomatosis With Polyangiitis | Wegener'sUnited States, Canada
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University of PennsylvaniaNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaboratorsCompletedTakayasu's ArteritisUnited States, Canada
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University of RochesterNational Institute of Neurological Disorders and Stroke (NINDS); Rare Diseases... and other collaboratorsTerminatedAndersen-Tawil SyndromeUnited States
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Children's Hospital Medical Center, CincinnatiNational Center for Research Resources (NCRR); Rare Diseases Clinical Research...CompletedPulmonary Alveolar ProteinosisUnited States, Germany, Italy, Japan
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Peter MerkelNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaboratorsCompletedTakayasu ArteritisUnited States, Canada
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University of South FloridaNational Institutes of Health (NIH); Rare Diseases Clinical Research NetworkCompletedOsteogenesis Imperfecta | Brittle Bone DisordersUnited States
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University of South FloridaNational Institutes of Health (NIH); Rare Diseases Clinical Research NetworkCompletedOsteogenesis Imperfecta | Brittle Bone DisordersUnited States
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AIDS Clinical Trials GroupNational Institute of Allergy and Infectious Diseases (NIAID); Neurologic AIDS...TerminatedHIV InfectionsUnited States
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompleted
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Icahn School of Medicine at Mount SinaiNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Rare... and other collaboratorsCompletedErythropoietic Protoporphyria | EPP | XLP | X-Linked Protoporphyria | XLPP | X-Linked Dominant Erythropoietic Protoporphyria | XLEPP | XLDPUnited States
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University of CincinnatiNational Heart, Lung, and Blood Institute (NHLBI); Rare Diseases Clinical Research...CompletedPulmonary Langerhans Cell HistiocytosisUnited States
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University of CincinnatiNational Heart, Lung, and Blood Institute (NHLBI); Rare Diseases Clinical Research...CompletedBirt-Hogg-Dube SyndromeUnited States
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National Center for Research Resources (NCRR)Mayo ClinicCompletedPolyradiculoneuropathy, Chronic Inflammatory Demyelinating
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National Center for Research Resources (NCRR)Scripps ClinicCompleted
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University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedObesity | Prader-Willi SyndromeUnited States
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CHU de Quebec-Universite LavalOttawa Hospital Research Institute; Canadian Critical Care Trials Group; Canadian...RecruitingBrain Injuries | Anemia | Traumatic Brain Injury | Subarachnoid Hemorrhage | Intensive Care Neurological Disorder | Near-Infrared SpectroscopyCanada
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS); Rare Diseases...RecruitingPyruvate Dehydrogenase Complex Deficiency DiseaseUnited States
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Children's Hospital Medical Center, CincinnatiNational Heart, Lung, and Blood Institute (NHLBI); Rare Diseases Clinical Research...CompletedAutoimmune Pulmonary Alveolar ProteinosisUnited States
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Children's Hospital Medical Center, CincinnatiRare Diseases Clinical Research Network; National Center for Advancing Translational...CompletedAutoimmune Pulmonary Alveolar ProteinosisUnited States
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CHU de Quebec-Universite LavalCanadian Institutes of Health Research (CIHR); Canadian Critical Care Trials... and other collaboratorsActive, not recruitingTraumatic Brain Injury | TransfusionCanada, United Kingdom, Brazil, France
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Brendan LeeEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedAmino Acid Metabolism, Inborn Errors | Urea Cycle Disorders | Argininosuccinic AciduriaUnited States
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Omar JawdatRare Diseases Clinical Research Network; Terra Biological LLC; Clinical Research...Unknown
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Icahn School of Medicine at Mount SinaiEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedApparent Mineralocorticoid Excess SyndromeUnited States, Brazil, France
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University of North Carolina, Chapel HillNational Institutes of Health (NIH); National Center for Research Resources... and other collaboratorsCompletedCystic Fibrosis | Primary Ciliary Dyskinesia | Kartagener Syndrome | PseudohypoaldosteronismUnited States, Canada
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National Human Genome Research Institute (NHGRI)Therapeutics for Rare and Neglected Diseases (TRND)RecruitingGNE Myopathy | GNE Related DiseasesUnited States
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Mark BatshawEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsTerminatedUrea Cycle DisordersUnited States, Canada
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University of PennsylvaniaNational Heart, Lung, and Blood Institute (NHLBI); National Institute of Arthritis... and other collaboratorsActive, not recruitingGranulomatosis With PolyangiitisUnited States, Canada
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedNiemann-Pick Disease, Type CUnited States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGaucher Disease Type 1United States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Late Onset Tay-Sachs DiseaseUnited States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedMucopolysaccharidosis Type IUnited States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsTerminatedTay-Sachs Disease | Sandhoff Disease | GM1 Gangliosidoses | GM2 GangliosidosesUnited States