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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Cerliponase Alfa Observational Study in the USCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative diseaseActive, not recruitingNot stated2026-06-30NCT04476862
Ensuring Access to Optimal Therapy in CF: The ENACT StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingPhase 42026-06-02NCT07148739
IBP-9414 for the Prevention of Necrotizing Enterocolitis - The Connection StudyDigestive System Diseases, digestive system disorder, enterocolitis, gastroenteritis, intestinal disorder, necrotizing enterocolitisActive, not recruitingPhase 32024-04-12NCT03978000