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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, erythropoietic protoporphyria, hepatic porphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, porphyria, skin disorderRecruitingNot stated2026-05-05NCT07567131
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, amyloidosis, cardiomyopathy, cardiovascular disorder, familial amyloid neuropathy, heart disorder, hereditary amyloidosis, hereditary disease, inborn errors of metabolism, metabolic disease, proteostasis deficienciesRecruitingPhase 32026-03-27NCT06128629