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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive CrisisCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Male Urogenital Diseases, Respiration Disorders, Urogenital Diseases, Vaso-Occlusive Crises, acute chest syndrome, acute kidney injury, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, kidney disorder, kidney failure, lung disorder, respiratory system disorder, sickle cell disease, urinary system disorderRecruitingPhase 22026-10-08NCT06699849
HCRN Endoscopic Versus Shunt Treatment of Hydrocephalus in Infantsbrain disorder, central nervous system disorder, hydrocephalus, nervous system disorderActive, not recruitingNot applicable2026-09-25NCT04177914
ACT001 for the Treatment of Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade GliomasNeoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, diffuse intrinsic pontine glioma, epithelial neoplasm, glioma, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingPhase 22026-09-21NCT06838676
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
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated CardiomyopathyCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, laminopathy, restrictive cardiomyopathy, subvalvular aortic stenosisActive, not recruitingNot stated2026-09-02NCT05112237
PEP-CMV Vaccine Targeting CMV Antigen to Treat Newly Diagnosed Pediatric HGG and DIPG and Recurrent MedulloblastomaNeoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, diffuse intrinsic pontine glioma, epithelial neoplasm, glioma, malignant glioma, medulloblastoma, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingPhase 22026-08-24NCT05096481
A-LiNK: Improving Outcomes in Autoimmune Liver DiseaseCholangitis, Sclerosing, Digestive System Diseases, Hepatic Insufficiency, Hepatitis, Chronic, autoimmune disease, autoimmune hepatitis, bile duct disorder, biliary tract disorder, cholangitis, chronic liver failure, hepatitis, immune system disorder, liver disorder, liver failure, primary sclerosing cholangitisRecruitingNot stated2026-08-19NCT05750498
TReatment for ImmUne Mediated PathopHysiologyBrain Diseases, Metabolic, Digestive System Diseases, Hepatic Insufficiency, Nutritional and Metabolic Diseases, acute liver failure, brain disorder, central nervous system disorder, hepatic encephalopathy, liver disorder, liver failure, metabolic disease, nervous system disorderActive, not recruitingPhase 22026-08-12NCT04862221
Comparative Effectiveness of Palliative Surgery Versus Additional Anti-Seizure Medications for Lennox-Gastaut SyndromeCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Epileptic Syndromes, Lennox-Gastaut syndrome, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, hereditary disease, nervous system disorder, visual epilepsyRecruitingNot stated2026-08-06NCT05374824
COMPASSION S3 - Evaluation of the SAPIEN 3 Transcatheter Heart Valve in Patients With Pulmonary Valve DysfunctionCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Pulmonary Atresia, Ventricular Outflow Obstruction, aortic valve disorder, aortopulmonary window, cardiovascular disorder, congenital heart disease, heart disorder, heart septal defect, heart valve disorder, persistent truncus arteriosus, pulmonary valve insufficiency, pulmonary valve stenosis, tetralogy of fallot, transposition of the great arteries, vascular malformationRecruitingNot applicable2026-07-23NCT02744677
Safety Study of Viaskin® Peanut Patch in Peanut-Allergic Children 1 Through 3 Years of Age (COMFORT Toddlers)Hypersensitivity, Immediate, Nut and Peanut Hypersensitivity, Peanut Hypersensitivity, allergic disease, food allergy, immune system disorderActive, not recruitingPhase 32026-07-22NCT07003919
A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's DisorderCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Tourette syndrome, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, neurodevelopmental disorder, psychiatric disorder, tic disorderActive, not recruitingPhase 32026-07-22NCT06021522
ARDS in Children and ECMO Initiation Strategies Impact on Neurodevelopment (ASCEND)Respiration Disorders, adult acute respiratory distress syndrome, lung disorder, respiratory system disorderActive, not recruitingNot stated2026-07-14NCT05388708
Rethinking Early Airway Clearence TherapiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderNot yet recruitingNot applicable2026-07-10NCT07696338
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young ChildrenCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingNot stated2026-07-08NCT04509050
Advanced SPinal Innovations With Robotics and Enabling Technology Registryadolescent idiopathic scoliosis, bone disorder, idiopathic scoliosis, musculoskeletal system disorder, spondylolisthesis, spondylolysis, spondylosis, vertebral column disorderRecruitingNot stated2026-06-22NCT05071144
DAY101 vs. Standard of Care Chemotherapy in Pediatric Participants With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)Noonan syndrome 5, low grade gliomaActive, not recruitingPhase 32026-05-14NCT05566795
HCRN Core Data Project: Characterizing Patient Populations in the Hydrocephalus Clinical Research Network (HCRN)Pediatric HydrocephalusRecruitingNot stated2026-05-14NCT00670735
AVATAR Anesthesia Alternative in Pediatric Radiotherapy: A Multi-Center StudyAnesthesiaActive, not recruitingNot stated2026-04-13NCT03991156
Azithromycin Treatment for Respiratory Syncytial Virus-induced Respiratory Failure in ChildrenInfections, Mononegavirales infectious disease, Paramyxoviridae infectious disease, Pneumovirus Infections, RNA Virus Infections, respiratory syncytial virus infectious disease, viral infectious diseaseRecruitingPhase 32026-02-09NCT05026749
COMPASSION S3 Post-Approval StudyBites and Stings, Chemically-Induced Disorders, cardiovascular disorder, heart disorder, heart valve disorder, injury, poisoning, pulmonary valve insufficiencyActive, not recruitingNot stated2026-02-03NCT04860765
Follow-up Automatically vs. As-Needed Comparison (FAAN-C) TrialDigestive System Diseases, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Infections, Male Urogenital Diseases, Soft Tissue Infections, Urogenital Diseases, digestive system disorder, gastroenteritis, lung disorder, pneumonia, respiratory system disorder, respiratory tract infectious disorder, urinary system disorder, urinary tract infectionRecruitingNot applicable2025-12-10NCT05471908
CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical OutcomesCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingNot stated2025-10-10NCT03350828
Quality of Pediatric Resuscitation in a Multicenter CollaborativeCardiopulmonary Arrest, cardiac arrest, cardiovascular disorder, heart disorderRecruitingNot stated2025-10-02NCT02708134
ALTERRA: SAPIEN 3 THV With the Alterra Adaptive PrestentCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorder, heart valve disorder, lung disorder, pulmonary valve insufficiency, respiratory system disorder, tetralogy of fallotActive, not recruitingNot applicable2025-09-25NCT03130777