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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Ziftomenib in Combination With Chemotherapy for Children With Relapsed/Refractory Acute LeukemiaDisease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, RecurrenceRecruitingPhase 12026-10-09NCT06376162
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
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
Metabolic Mechanisms Induced by Enteral DHA and ARA Supplementation in Preterm InfantsFemale Urogenital Diseases and Pregnancy Complications, Obstetric Labor Complications, Obstetric Labor, Premature, Premature Birth, Urogenital Diseases, pregnancy disorderRecruitingNot applicable2026-10-07NCT05380401
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
Postoperative Pacing for Cardiac EfficiencyCardiac Pacing; Pacing Therapy; Pediatric Congenital Heart Surgery; Postoperative Cardiac Surgery; Postoperative CareRecruitingNot applicable2026-10-01NCT07852390
A Study to Find Out if BI 764198 Helps Adults and Adolescents With a Kidney Condition Called Focal Segmental Glomerulosclerosis (FSGS)Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Urogenital Diseases, focal segmental glomerulosclerosis, glomerulonephritis, kidney disorder, nephritis, urinary system disorderRecruitingPhase 32026-09-30NCT07220083
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Conditioncongenital structural myopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseRecruitingNot stated2026-09-30NCT06581146
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC DeficiencyBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Hyperammonemia, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, liver disorder, metabolic disease, nervous system disorder, ornithine carbamoyltransferase deficiency, urea cycle disorderActive, not recruitingPhase 32026-09-30NCT06255782
PODOMOUNT-Basket, a Study to Test Whether BI 764198 Helps Adults and Adolescents With Different Types of Kidney DiseaseProteinuric Kidney DiseasesRecruitingPhase 22026-09-29NCT07355296
Multicenter Study of Lumateperone for the Treatment of Bipolar Depression in Pediatric PatientsBipolar and Related Disorders, bipolar disorder, mood disorder, psychiatric disorderRecruitingPhase 32026-09-25NCT06372964
A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)Bone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderActive, not recruitingPhase 32026-09-25NCT05600426
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With AchondroplasiaBone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasiaRecruitingPhase 2 / Phase 32026-09-24NCT07441876
Bowel Continence Across the Lifespan in People With Spina BifidaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Fecal Incontinence, central nervous system malformation, colonic disorder, digestive system disorder, functional colonic disease, intestinal disorder, nervous system disorder, neurogenic bowel, rectal disorder, spina bifidaRecruitingNot stated2026-09-24NCT07390318
A Study of Tirzepatide in Adolescents With Obesity and Weight-Related Comorbidities (SURMOUNT-ADOLESCENTS-2)Body Weight, Body Weight Changes, Nutritional and Metabolic Diseases, Overweight, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Weight Gain, nutritional disorder, obesity disorder, overnutritionRecruitingPhase 32026-09-24NCT06439277
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingPhase 1 / Phase 22026-09-24NCT06270316
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone TreatmentBone Diseases, Developmental, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Dwarfism, Musculoskeletal Abnormalities, Noonan syndrome, Skin and Connective Tissue Diseases, bone disorder, cardiovascular disorder, congenital heart disease, connective tissue disorder, endocrine system disorder, heart disorder, hereditary disease, musculoskeletal system disorderActive, not recruitingPhase 22026-09-23NCT06668805
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, generalized epilepsy with febrile seizures plus, type 2, nervous system disorderRecruitingPhase 1 / Phase 22026-09-22NCT05419492
Caring Contacts Via Text Message for Suicidal Adolescents After Emergency Department DischargeSuicide PreventionRecruitingNot applicable2026-09-21NCT07043764
A Study to Find Out How EMPAgliflozin is Tolerated and if it Helps Children and Adolescents With Chronic KIDNEY Disease (EMPA-KIDNEY® Kids)Chronic Disease, Disease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, chronic renal failure syndrome, kidney disorder, kidney failure, urinary system disorderRecruitingPhase 32026-09-16NCT07107945
Comparative Effectiveness of SPARCS and Unified Protocol for Youth With Chronic Medical Conditions and Trauma-Related DistressStress Disorders, Traumatic, acute stress disorder, injury, post-traumatic stress disorder, psychiatric disorderRecruitingNot applicable2026-09-15NCT07483333
Post-Vent, the Sequelae: Personalized Prognostic Modeling for Consequences of Neonatal Intermittent Hypoxemia in Preterm Infants at Pre-School AgeApnea, Dyssomnias, Female Urogenital Diseases and Pregnancy Complications, Obstetric Labor Complications, Obstetric Labor, Premature, Premature Birth, Respiration Disorders, Sleep Disorders, Intrinsic, Urogenital Diseases, nervous system disorder, neurodevelopmental disorder, pregnancy disorder, psychiatric disorder, respiratory system disorder, sleep apnea syndrome, sleep-wake disorderRecruitingNot stated2026-09-15NCT05336890
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, nervous system disorder, visual epilepsyRecruitingPhase 32026-09-15NCT04462770
Determine Maximum Tolerated Dose, Safety, and Tolerability of Rhenium (186Re) in Pediatric Recurrent, Refractory or Progressive Ependymoma and High-Grade GliomaNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, ependymoma, epithelial neoplasm, glioma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingPhase 12026-09-11NCT07061626
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory CohortCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseRecruitingPhase 12026-09-11NCT04626674