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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorderActive, not recruitingPhase 32026-10-08NCT06634420
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic DystrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular diseaseRecruitingPhase 2 / Phase 32025-05-28NCT05004129