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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Efficacy and Safety of Zelpultide Alfa in Preterm Neonates at High Risk of Developing Bronchopulmonary Dysplasia (BPD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infant, Newborn, Diseases, Infant, Premature, Diseases, Lung Injury, Ventilator-Induced Lung Injury, bronchopulmonary dysplasia, lung disorder, respiratory system disorderRecruitingPhase 2 / Phase 32026-10-09NCT06897839
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 Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderActive, not recruitingPhase 32026-10-02NCT04573023
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 DeficiencyCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, arterial calcification of infancy, autosomal recessive hypophosphatemic rickets, autosomal recessive inherited pseudoxanthoma elasticum, cardiovascular disorder, connective tissue disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary skin disorder, hypophosphatemic rickets, autosomal recessive, 1, skin disorder, vascular disorder, vascular hemostatic diseaseActive, not recruitingPhase 12026-09-29NCT05734196
Decoding Personalized Nutritional, Microbiome and Host Patterns Impacting Clinical and Prognostic Features in Crohn's DiseaseCrohn disease, Digestive System Diseases, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorderActive, not recruitingNot stated2026-09-29NCT04283864
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
Safety and Efficacy of OC-1 Therapy in Patients With R/R T-ALL/LLHemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, T-cell acute lymphoblastic leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasmRecruitingPhase 12026-03-11NCT05679895
An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) SubjectsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderEnrolling by invitationPhase 32026-01-21NCT05594992
European and North Indian Cohort of KaWasaki dIseaseHemic and Lymphatic Diseases, Kawasaki disease, Skin and Connective Tissue Diseases, cardiovascular disorder, lymphatic system disorder, skin disorder, skin vascular disease, vascular disorder, vasculitisRecruitingNot stated2025-09-29NCT06305611
Vyxeos® With Clofarabine for Pediatric AMLDisease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, RecurrenceRecruitingPhase 12025-09-05NCT07156435
sFlt1/PlGF and Planned Delivery to Prevent Preeclampsia at Term.Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Female Urogenital Diseases and Pregnancy Complications, Fetal Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Perinatal Death, Urogenital Diseases, cardiovascular disorder, fetal growth restriction, hereditary endocrine growth disease, hypertension, pregnancy-induced, hypertensive disorder, preeclampsia, pregnancy disorder, vascular disorderRecruitingNot applicable2025-02-19NCT04766866