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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Rett syndrome, X-linked disease, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorderRecruitingPhase 1 / Phase 22026-10-08NCT06430385
A Study to Assess Sulbactam-durlobactam in Pediatric Patients With Acinetobacter Baumannii-calcoaceticus Complex InfectionAcinetobacter Baumannii-calcoaceticus Complex Infection (ABC)RecruitingPhase 12026-10-06NCT06801223
Silmitasertib (CX-4945) in Combination With Chemotherapy for Relapsed Refractory Solid TumorsEwing sarcoma, Myosarcoma, Neoplasms by Histologic Type, Neoplasms, Adipose Tissue, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, connective tissue neoplasm, epithelial neoplasm, liposarcoma, myomatous neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, primitive neuroectodermal tumor, rhabdomyosarcoma, sarcomaRecruitingPhase 1 / Phase 22026-10-06NCT06541262
ALTERRA Post-Approval StudyCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorder, heart valve disorder, pulmonary valve insufficiency, tetralogy of fallotActive, not recruitingNot stated2026-10-05NCT05378386
Fimepinostat in Treating Brain Tumors in Children and Young AdultsNeoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, astrocytoma (excluding glioblastoma), brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, diffuse intrinsic pontine glioma, epithelial neoplasm, glioblastoma, glioma, medulloblastoma, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingEarly phase 12026-10-05NCT03893487
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille SyndromeAbnormalities, Multiple, Alagille syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, bile duct disorder, biliary tract disorder, cardiovascular disorder, cholestasis, congenital heart disease, hereditary disease, intrahepatic cholestasis, liver disorderActive, not recruitingPhase 32026-10-02NCT05035030
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromeAbnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorderActive, not recruitingPhase 32026-09-29NCT06914609
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 DeficiencyCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, arterial calcification of infancy, autosomal recessive hypophosphatemic rickets, autosomal recessive inherited pseudoxanthoma elasticum, cardiovascular disorder, connective tissue disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary skin disorder, hypophosphatemic rickets, autosomal recessive, 1, skin disorder, vascular disorder, vascular hemostatic diseaseActive, not recruitingPhase 12026-09-29NCT05734196
A Study of Orforglipron (LY3502970) Compared With Dulaglutide in Pediatric Participants With Type 2 DiabetesNutritional and Metabolic Diseases, diabetes mellitus, endocrine system disorder, glucose metabolism disease, metabolic disease, type 2 diabetes mellitusNot yet recruitingPhase 32026-09-25NCT07668336
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
BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorderRecruitingPhase 32026-09-10NCT07605429
A Study to Assess the Efficacy and Safety of Daily OM-85 in Young Children With Recurrent WheezingPathological Conditions, Signs and Symptoms, Respiratory Sounds, Signs and Symptoms, Signs and Symptoms, RespiratoryActive, not recruitingPhase 22026-09-08NCT05857930
Pediatric Precision Laboratory Advanced Neuroblastoma TherapyNeoplasms 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 tumorRecruitingPhase 22026-09-04NCT02559778
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
Neuroblastoma Maintenance Therapy TrialNeoplasms 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 22026-09-03NCT02679144
Phase 2 Study of Alisertib Therapy for Rhabdoid TumorsNeoplasms by Histologic Type, atypical teratoid rhabdoid tumor, mixed neoplasm, neoplasm, rhabdoid tumorActive, not recruitingPhase 22026-09-01NCT02114229
Non-invasive Prediction of EArly Cardiac rEjectionCardiac MRI; Cell Free DNA; Heart Transplant; Pediatric Heart Transplant; RejectionRecruitingNot stated2026-08-20NCT07770659
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman SyndromeAbnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorderActive, not recruitingPhase 1 / Phase 22026-08-20NCT05127226
Naxitamab Added to Induction for Newly Diagnosed High-Risk 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 tumorRecruitingPhase 22026-08-12NCT05489887
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
DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed MedulloblastomaNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, epithelial neoplasm, glioma, medulloblastoma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingPhase 22026-08-12NCT04696029
Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma and EpendymomaMedulloblastoma, Childhood, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, ependymoma, epithelial neoplasm, glioma, medulloblastoma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingNot applicable2026-08-11NCT05057702
A Phase 3 Trial to Compare IV BCV Versus IV CDV for Treatment of Adenovirus Infection After Allo-HCTDNA Virus Infections, Infections, adenoviridae infectious disease, viral infectious diseaseRecruitingPhase 32026-08-10NCT07387367
Adding Dasatinib Or Venetoclax To Improve Responses In Children With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (ALL) Or Lymphoma (T-LLY) Or Mixed Phenotype Acute Leukemia (MPAL)Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, T-cell acute lymphoblastic leukemia, T-cell non-Hodgkin lymphoma, acute biphenotypic leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, mixed phenotype acute leukemia, neoplasmRecruitingPhase 22026-08-10NCT06390319
A Trial of Setmelanotide in Acquired Hypothalamic ObesityCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Froelich syndrome, Male Urogenital Diseases, Urogenital Abnormalities, Urogenital Diseases, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, hypogonadismActive, not recruitingPhase 32026-08-10NCT05774756