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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Find Out How Nerandomilast is Tolerated, Handled by the Body, and if it Helps Children and Adolescents With Interstitial Lung Disease (FIBRONEER-chILD)Fibrosing Interstitial Lung DiseaseRecruitingPhase 32026-09-29NCT07366034
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosisRecruitingPhase 32026-09-28NCT07082725
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 GangliosidosisBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM1 gangliosidosis, GM2 gangliosidosis, Gangliosidoses, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosisRecruitingPhase 32026-09-24NCT07054515
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 GangliosidosisBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM1 gangliosidosis, GM2 gangliosidosis, Gangliosidoses, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosisActive, not recruitingPhase 32026-09-22NCT07082543
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 3ARecruitingPhase 2 / Phase 32026-09-22NCT02716246
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorderActive, not recruitingPhase 32026-09-17NCT03911505
Pediatric Influence of Cooling Duration on Efficacy in Cardiac Arrest Patients (P-ICECAP)Body Temperature Changes, Coma, Consciousness Disorders, Hypothermia, Hypoxia, Hypoxia, Brain, Neurobehavioral Manifestations, Neurologic Manifestations, Out-of-Hospital Cardiac Arrest, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Signs and Symptoms, Respiratory, Unconsciousness, brain disorder, brain hypoxia - ischemia, brain ischemia, cardiac arrest, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, heart disorder, nervous system disorder, vascular disorderRecruitingNot applicable2026-09-08NCT05376267
Minimising Adverse Drug Reactions and Verifying Economic Legitimacy in Children (MARVEL-PIC)neoplasmActive, not recruitingNot applicable2026-07-29NCT05667766
Immunotherapy Before and After Surgery for Treatment of Recurrent or Progressive High Grade Glioma in Children and Young AdultsNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, astrocytoma (excluding glioblastoma), epithelial neoplasm, glioblastoma, glioma, grade III glioma, malignant glioma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingPhase 12026-06-15NCT04323046
Staphylococcus Aureus Network Adaptive Platform TrialBacterial Infections and Mycoses, Infections, bacterial infectious disease, gram-positive bacterial infections, staphylococcal infectionRecruitingPhase 42026-05-06NCT05137119
A Study to Test the Safety and Efficacy of the Drug Larotrectinib for the Treatment of Tumors With NTRK-fusion in ChildrenneoplasmActive, not recruitingPhase 1 / Phase 22025-06-22NCT02637687
Antenatal Melatonin Supplementation for Neuroprotection in Fetal Growth RestrictionCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Female Urogenital Diseases and Pregnancy Complications, Fetal Death, Fetal Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, fetal growth restriction, hereditary endocrine growth disease, pregnancy disorder, pregnancy disorder with abortive outcomeRecruitingPhase 32025-06-06NCT05651347
International Chronic Myeloid Leukemia Pediatric StudyChronic Disease, Disease Attributes, Hemic and Lymphatic Diseases, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myeloproliferative Disorders, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, bone marrow disorder, hematologic disorder, leukemia, myeloid leukemia, neoplasmRecruitingNot stated2024-01-17NCT01281735