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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
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 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
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 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 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
Ketamine add-on Therapy for Established Status Epilepticus Treatment Trial (KESETT)Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Status Epilepticus, nervous system disorder, visual epilepsyRecruitingPhase 32026-09-17NCT06907173
Eflornithine (DFMO) and Etoposide for Relapsed/Refractory NeuroblastomaNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, epithelial neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingPhase 22026-09-03NCT04301843
Conditioning SCID Infants Diagnosed Earlysevere combined immunodeficiencyActive, not recruitingPhase 22026-08-28NCT03619551
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
Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Malformations of Cortical Development, Group I, Muscle Hypertonia, Muscle Spasticity, Musculoskeletal Abnormalities, Neurologic Manifestations, Neuromuscular Manifestations, Paraplegia, Spastic, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, central nervous system malformation, cerebral cortical dysplasia, hereditary spastic paraplegia 50, microcephaly, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, palsy, paraplegiaRecruitingPhase 1 / Phase 22026-07-08NCT05518188
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)Bone Marrow Failure Disorders, Chronic Disease, Disease Attributes, Hemic and Lymphatic Diseases, Hodgkins lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myeloproliferative Disorders, Neoplasms by Histologic Type, Neoplasms by Site, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, acute lymphoblastic leukemia, acute myeloid leukemia, anemia, aplastic anemia, autoimmune disease, bone marrow disorder, chronic myeloid leukemia, disease, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, immunoproliferative disorder, inborn errors of metabolism, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, myeloid leukemia, neoplasm, non-Hodgkin lymphomaRecruitingNot stated2026-07-06NCT01351545
QUELIMMUNE (SCD-PED) PediAtric SurVeillance REgistryFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Inflammation, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, acute kidney injury, kidney disorder, kidney failure, urinary system disorderRecruitingNot stated2026-05-14NCT06517810
A Long-Term Study of JNT-517 in Participants With PhenylketonuriaBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuriaRecruitingPhase 32026-05-01NCT06628128
International Study of Cerebral Oxygenation and Electrical Activity During Major Neonatal SurgeryCongenital Disorders, Congenital, Hereditary, and Neonatal Diseases and AbnormalitiesRecruitingNot stated2026-04-23NCT05673499
SJM Masters HP 15mm Rotatable Mechanical Heart Valve as Aortic Valve Replacement Therapyaortic valve disorder, cardiovascular disorder, heart disorder, heart valve disorderActive, not recruitingNot stated2026-04-13NCT03924661
Eliminating Monitor Overuse Trial (EMO Trial)Bronchiolitis Acute ViralActive, not recruitingNot applicable2026-02-13NCT05132322
Social & Contextual Impact on Children Undergoing Liver TransplantationDigestive System Diseases, liver disorderActive, not recruitingNot stated2026-01-26NCT04551742
The Pediatric Anesthesia Quality Improvement ProjectAnesthesia; Children; SurgeryRecruitingNot stated2025-05-21NCT00674388