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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Learn About the Effects of Felzartamab Infusions on Adults With Immunoglobulin A Nephropathy (IgAN)Female 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 32026-09-30NCT06935357
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring ProgramCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, disorder of glycogen metabolism, glucose metabolism disease, glycogen storage disease I, glycogen storage disease due to glucose-6-phosphatase deficiency type IA, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, metabolic diseaseRecruitingNot stated2026-08-26NCT06636383
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseActive, not recruitingPhase 2 / Phase 32026-06-23NCT05031780
A Study to Evaluate the Safety and Tolerability, Pharmacokinetics, and Antiviral Activity of Maribavir for the Treatment of Cytomegalovirus (CMV) Infection in Children and Adolescents Who Have Received a Hematopoietic Stem Cell Transplant (HSCT) or a Solid Organ Transplant (SOT)Cytomegalovirus (CMV)RecruitingPhase 32026-05-22NCT05319353
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical PracticeCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Respiration Disorders, cardiomyopathy, cardiovascular disorder, heart disorder, hereditary disease, limb-girdle muscular dystrophy, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease, respiratory system disorder, sarcoglycanopathyActive, not recruitingNot stated2025-11-06NCT04475926