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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
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
CACP: Study on Camptodactyly - Arthropathy - Coxa Vara - Pericarditis (CACP) SyndromeBone Anteversion, Bone Malalignment, Pathologic Processes, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Torsion Abnormality, arthropathy, bone disorder, cardiovascular disorder, coxa vara, disease, heart disorder, musculoskeletal system disorder, pericarditis, syndromic diseaseRecruitingNot stated2026-03-16NCT07468461
Multicenter Italian Cohort Study on Tuberculosis in Pediatric AgeActinomycetales infectious disease, Bacterial Infections and Mycoses, Infections, bacterial infectious disease, disease arising from reactivation of latent virus, gram-positive bacterial infections, latent tuberculosis infection, mycobacterial infectious disease, tuberculosisRecruitingNot stated2024-03-04NCT06289660