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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 22026-10-08NCT07287982
Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, hematologic disorder, leukemia, myeloid leukemia, neoplasmRecruitingPhase 32026-08-24NCT05183035
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)Abnormalities, Multiple, Arrhythmias, Cardiac, COVID-19, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Coronaviridae Infections, Down syndrome, Dyssomnias, Edema, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Hemic and Lymphatic Diseases, Hemorrhage, Infant, Newborn, Diseases, Infant, Premature, Diseases, Infections, Inflammation, Kawasaki disease, Lung Injury, Menorrhagia, Menstruation Disturbances, Neurobehavioral Manifestations, Neurologic Manifestations, Nidovirales infectious disease, Nutritional and Metabolic Diseases, Orthocoronavirinae infectious disease, Pain, Pathologic Processes, Pathological Conditions, Signs and Symptoms, RNA Virus Infections, Signs and Symptoms, Skin and Connective Tissue Diseases, Sleep Disorders, Intrinsic, Suppuration, Urogenital Diseases, Uterine Hemorrhage, Ventilator-Induced Lung Injury, Water-Electrolyte Imbalance, adrenal gland disorder, adrenal gland hyperfunction, adrenocortical insufficiency, attention deficit hyperactivity disorder, inattentive type, blood coagulation disease, bronchopulmonary dysplasia, cardiovascular disorder, cellulitis, chromosomal disorder, coagulation protein disease, connective tissue disorder, endocrine system disorder, female reproductive system disorder, heart disorder, heart failure, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, hyperaldosteronism, hyperphosphatemia, hypertensive disorder, inherited blood coagulation disorder, insomnia, intellectual disability, lung disorder, lymphatic system disorder, metabolic disease, nervous system disorder, neurodevelopmental disorder, oppositional defiant disorder, pediatric multisystem inflammatory disease, COVID-19 related, phosphorus metabolism disease, pneumonia, potassium deficiency disease, primary aldosteronism, psychiatric disorder, pulmonary arterial hypertension, pulmonary hypertension, respiratory system disorder, respiratory tract infectious disorder, skin disorder, skin disorder caused by infection, skin infection, skin vascular disease, sleep-wake disorder, uterine disorder, vascular disorder, vascular hemostatic disease, vasculitis, viral infectious disease, viral pneumoniaRecruitingNot stated2026-08-21NCT04278404
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular DystrophyMuscular dystrophy congenital, merosin negativeRecruitingNot stated2026-08-19NCT06503367
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)Congenital, 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 3AActive, not recruitingPhase 1 / Phase 22026-07-13NCT06181136
Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9autosomal recessive limb-girdle muscular dystrophy type 2IActive, not recruitingPhase 32026-07-02NCT07678775
A Phase 2 Study of Vosoritide in Children With Idiopathic Short StatureIdiopathic Short StatureRecruitingPhase 22026-05-22NCT06382155
Quality of Pediatric Resuscitation in a Multicenter CollaborativeCardiopulmonary Arrest, cardiac arrest, cardiovascular disorder, heart disorderRecruitingNot stated2025-10-02NCT02708134
Interactive Resuscitation Application for Mothers About to Experience Premature Infant ResuscitationResuscitationRecruitingNot stated2025-08-12NCT05243043
The Pediatric Anesthesia Quality Improvement ProjectAnesthesia; Children; SurgeryRecruitingNot stated2025-05-21NCT00674388