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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Study of Re-Treatment With ALXN2220 in Patients With ATTR-CMNutritional and Metabolic Diseases, amyloidosis, metabolic disease, proteostasis deficienciesActive, not recruitingPhase 22026-10-07NCT07213583
Study of Ravulizumab in Immunoglobulin A Nephropathy (IgAN)Chronic Disease, Disease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, IgA glomerulonephritis, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Urination Disorders, Urogenital Diseases, Urological Manifestations, autoimmune disease, chronic renal failure syndrome, glomerulonephritis, immune system disorder, kidney disorder, kidney failure, nephritis, proteinuria, urinary system disorderActive, not recruitingPhase 32026-10-02NCT06291376
Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUSCytopenia, Embolism and Thrombosis, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, Intracranial Embolism and Thrombosis, Thromboembolism, anemia, bone marrow disorder, brain disorder, brain ischemia, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hematologic disorder, hemolytic anemia, intracranial thrombosis, myelodysplastic syndrome, nervous system disorder, paroxysmal nocturnal hemoglobinuria, transient ischemic attack, vascular disorderActive, not recruitingNot stated2026-09-18NCT03329365
Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG)Neoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, generalized myasthenia gravis, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndromeRecruitingPhase 32026-09-11NCT07596784
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD)CNS demyelinating autoimmune disease, acute transverse myelitis, autoimmune disease, autoimmune disorder of the nervous system, cranial nerve neuropathy, demyelinating disease, eye disorder, immune system disorder, nervous system disorder, neuromyelitis optica, optic nerve disorder, optic neuritisRecruitingPhase 32026-09-11NCT07557420
Registry of Patients With HypophosphatasiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, hereditary disease, hypophosphatasia, inborn errors of metabolism, inborn metal metabolism disorder, metabolic diseaseEnrolling by invitationNot stated2026-09-04NCT02306720
An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid CardiomyopathyTransthyretin Amyloid Cardiomyopathy (ATTR-CM)Not yet recruitingPhase 32026-08-28NCT07789483
ALXN2350 in Adult Participants With BAG3-Associated Dilated CardiomyopathyBAG3 Mutation Associated Dilated CardiomyopathyRecruitingPhase 1 / Phase 22026-08-28NCT07218887
Phase 2 Study of ALXN2030 in Patients With Antibody-Mediated Rejection After Kidney TransplantationAMR; Antibody-Mediated Rejection; Biopsy-proven Histologic Scores; Kidney TransplantationActive, not recruitingPhase 22026-08-11NCT06744647
Study of ALXN2220 Versus Placebo in Adults With ATTR-CMNutritional and Metabolic Diseases, amyloidosis, metabolic disease, proteostasis deficienciesActive, not recruitingPhase 32026-08-10NCT06183931
Study of Ravulizumab in Pediatric Participants With Primary IgANFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Hemorrhage, IgA glomerulonephritis, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, Urination Disorders, Urogenital Diseases, Urological Manifestations, autoimmune disease, blood coagulation disease, cardiovascular disorder, glomerulonephritis, hematologic disorder, hemorrhagic disease, immune system disorder, immunoglobulin A vasculitis, kidney disorder, nephritis, proteinuria, purpura, skin disorder, skin vascular disease, type III hypersensitivity disease, urinary system disorder, vascular disorder, vascular hemostatic disease, vasculitisRecruitingPhase 32026-07-30NCT07024563
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Ceroid Lipofuscinosis, Neuronal, 2, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Fabry disease, GM1 gangliosidosis, Gangliosidoses, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant, Newborn, Diseases, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mucopolysaccharidosis III, Neurobehavioral Manifestations, Neurologic Manifestations, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Skin and Connective Tissue Diseases, Sulfatidosis, Wolman disease, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrotendinous xanthomatosis, cerebrovascular disorder, cholesteryl ester storage disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, hereditary disease, histiocytosis, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis, vascular disorder, xanthomatosisEnrolling by invitationNot stated2026-07-28NCT05368038
Acceptability and Safety of MR-C-014 in Persons With Neuromyelitis Optica Spectrum DisorderCNS demyelinating autoimmune disease, acute transverse myelitis, autoimmune disease, autoimmune disorder of the nervous system, cranial nerve neuropathy, demyelinating disease, eye disorder, immune system disorder, nervous system disorder, neuromyelitis optica, optic nerve disorder, optic neuritisEnrolling by invitationNot applicable2026-07-06NCT06374264
A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.Immunoglobulin Light-chain Amyloidosis, Neoplasms by Histologic Type, Nutritional and Metabolic Diseases, amyloidosis, immune system disorder, immunoproliferative disorder, lymphoproliferative syndrome, metabolic disease, monoclonal gammopathy, neoplasm, plasma cell neoplasm, proteostasis deficienciesRecruitingPhase 1 / Phase 22026-07-02NCT07081646
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)Blood Protein Disorders, Hemic and Lymphatic Diseases, Immunoglobulin Light-chain Amyloidosis, Neoplasms by Histologic Type, Nutritional and Metabolic Diseases, amyloidosis, hematologic disorder, immune system disorder, immunoproliferative disorder, lymphoproliferative syndrome, metabolic disease, monoclonal gammopathy, neoplasm, plasma cell neoplasm, proteostasis deficienciesActive, not recruitingPhase 32026-07-02NCT04504825
Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSDCNS demyelinating autoimmune disease, acute transverse myelitis, autoimmune disease, autoimmune disorder of the nervous system, cranial nerve neuropathy, demyelinating disease, eye disorder, immune system disorder, nervous system disorder, neuromyelitis optica, optic nerve disorder, optic neuritisActive, not recruitingPhase 2 / Phase 32026-06-24NCT05346354
Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase AlfaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, hereditary disease, hypophosphatasia, inborn errors of metabolism, inborn metal metabolism disorder, metabolic diseaseActive, not recruitingPhase 32026-06-22NCT06079281
Study of ALXN1920 in Adult Participants With Primary Membranous Nephropathy (PMN)Primary Membranous NephropathyRecruitingPhase 22026-06-18NCT07157787
Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab Administered Intravenously in Adult Participants at High Risk of Delayed Graft Function After Kidney TransplantationDelayed Graft Function, Pathologic Processes, Pathological Conditions, Signs and SymptomsRecruitingPhase 32026-06-18NCT06830798
Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor TherapiesCNS demyelinating autoimmune disease, acute transverse myelitis, autoimmune disease, autoimmune disorder of the nervous system, cranial nerve neuropathy, demyelinating disease, eye disorder, immune system disorder, nervous system disorder, neuromyelitis optica, optic nerve disorder, optic neuritisRecruitingNot stated2026-06-18NCT05966467
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNHHemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, Hemolysis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingPhase 32026-06-18NCT05389449
Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia GravisNeoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndromeActive, not recruitingPhase 32026-06-17NCT05556096
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)Blood Protein Disorders, Hemic and Lymphatic Diseases, Immunoglobulin Light-chain Amyloidosis, Neoplasms by Histologic Type, Nutritional and Metabolic Diseases, amyloidosis, hematologic disorder, immune system disorder, immunoproliferative disorder, lymphoproliferative syndrome, metabolic disease, monoclonal gammopathy, neoplasm, plasma cell neoplasm, proteostasis deficienciesActive, not recruitingPhase 32026-06-17NCT04512235
Safety and Efficacy of Tarperprumig in Adult Participants With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated VasculitisSkin and Connective Tissue Diseases, anti-neutrophil cytoplasmic antibody-associated vasculitis, autoimmune disease, cardiovascular disorder, immune system disorder, necrotizing vasculitis, skin disorder, skin vascular disease, vascular disorder, vasculitisRecruitingPhase 22026-06-16NCT07160608
A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, hereditary disease, laminopathyActive, not recruitingNot stated2026-06-12NCT05981092