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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care TreatmentBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, brain disorder, central nervous system disorder, connective tissue disorder, hereditary disease, homocystinuria, hyperhomocysteinemia, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorderRecruitingPhase 32026-10-08NCT06247085
ETA and AT1 Antagonism in ANCA-vasculitis (SPARVASC)Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Skin and Connective Tissue Diseases, Urogenital Diseases, anti-neutrophil cytoplasmic antibody-associated vasculitis, autoimmune disease, cardiovascular disorder, immune system disorder, kidney disorder, necrotizing vasculitis, skin disorder, skin vascular disease, urinary system disorder, vascular disorder, vasculitisActive, not recruitingPhase 22026-08-26NCT05630612
Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, brain disorder, central nervous system disorder, connective tissue disorder, hereditary disease, homocystinuria, hyperhomocysteinemia, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorderRecruitingPhase 1 / Phase 22026-08-10NCT03406611
Sparsentan for the Treatment of VEGF Signaling Pathway Inhibitor-Associated ProteinuriaFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Urination Disorders, Urogenital Diseases, Urological Manifestations, neoplasm, proteinuria, urinary system disorderRecruitingPhase 12026-07-30NCT07224776
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
Sparsentan in Posttransplant Immunoglobulin A Nephropathy or Focal Segmental GlomerulosclerosisFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Urination Disorders, Urogenital Diseases, Urological Manifestations, focal segmental glomerulosclerosis, glomerulonephritis, kidney disorder, nephritis, proteinuria, urinary system disorderActive, not recruitingPhase 42026-06-23NCT07219121
A Study of the Safety and Activity of Sparsentan for the Treatment of Patients With Immunoglobulin A NephropathyFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, IgA glomerulonephritis, Male Urogenital Diseases, Urogenital Diseases, autoimmune disease, glomerulonephritis, immune system disorder, kidney disorder, nephritis, urinary system disorderRecruitingPhase 22026-06-03NCT04663204
A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, brain disorder, central nervous system disorder, connective tissue disorder, hereditary disease, homocystinuria, hyperhomocysteinemia, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorderEnrolling by invitationPhase 32026-05-28NCT06431893
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular DiseasesAlport syndrome, Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female 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, Urogenital Abnormalities, Urogenital Diseases, autoimmune disease, blood coagulation disease, cardiovascular disorder, connective tissue disorder, focal segmental glomerulosclerosis, glomerulonephritis, hematologic disorder, hemorrhagic disease, hereditary nephritis, immune system disorder, immunoglobulin A vasculitis, kidney disorder, macular corneal dystrophy, minimal change disease, nephritis, nephrosis, purpura, skin disorder, skin vascular disease, type III hypersensitivity disease, urinary system disorder, vascular disorder, vascular hemostatic disease, vasculitisRecruitingPhase 22026-05-12NCT05003986
A Study of the Effect and Safety of Sparsentan in the Treatment of Patients With IgA NephropathyFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, IgA glomerulonephritis, Male Urogenital Diseases, Urogenital Diseases, autoimmune disease, glomerulonephritis, immune system disorder, kidney disorder, nephritis, urinary system disorderActive, not recruitingPhase 32026-04-20NCT03762850