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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
An Efficacy and Safety Study of Clemizole HCl in Patients With 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 epilepsyRecruitingPhase 32026-10-09NCT05066217
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
Setrusumab vs Placebo for Osteogenesis ImperfectaBone Diseases, Developmental, Collagen Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia, osteogenesis imperfectaActive, not recruitingPhase 2 / Phase 32026-10-06NCT05125809
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
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)Congenital, 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 32026-09-28NCT07160634
Pyruvate Kinase Deficiency Global Longitudinal RegistryCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, congenital nonspherocytic hemolytic anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, pyruvate kinase deficiency of red cellsActive, not recruitingNot stated2026-09-28NCT03481738
A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseRecruitingPhase 32026-09-17NCT06612268
Massive Transfusion in Children-2: A Trial Examining Life Threatening Hemorrhage in ChildrenAccidental Injuries, Hemorrhage, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Shock, Shock, Hemorrhagic, injuryRecruitingPhase 32026-09-17NCT06070350
Open-Label Extension of EDG-5506 in Participants With Becker Muscular DystrophyBecker muscular dystrophy, Congenital, 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 diseaseEnrolling by invitationPhase 22026-09-17NCT06066580
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
Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, blood platelet disease, coagulation protein disease, hematologic disorder, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, von Willebrand disease (hereditary or acquired)RecruitingPhase 32026-09-14NCT07115004
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
A Study of Patients With Fabry Disease (US Specific)Brain 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 disorderRecruitingNot stated2026-09-09NCT06906367
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Inflammation, Muscular Disorders, Atrophic, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, bone fracture, hereditary disease, injury, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseActive, not recruitingPhase 22026-09-04NCT06450639
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
Tipifarnib and Naxitamab 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 tumorRecruitingPhase 22026-09-03NCT06540963
Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and SarcomasEwing sarcoma, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, atypical teratoid rhabdoid tumor, brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, connective tissue neoplasm, diffuse intrinsic pontine glioma, embryonal tumor with multilayered rosettes, epithelial neoplasm, glioma, mixed neoplasm, neoplasm, nervous system disorder, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, primitive neuroectodermal tumor, rhabdoid tumor, sarcomaRecruitingPhase 1 / Phase 22026-09-03NCT06465199
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
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
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular DystrophyCongenital, 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 diseaseActive, not recruitingPhase 32026-08-28NCT05126758
A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation (The ReInspire Study)CLOVES syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lymphatic Abnormalities, angiomatosis, angioosteohypertrophic syndrome, cardiovascular disorder, lymphatic system disorder, megalencephaly-capillary malformation-polymicrogyria syndrome, vascular disorder, vascular malformationRecruitingPhase 22026-08-25NCT06789913
A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, nervous system disorderRecruitingPhase 32026-08-24NCT06872125
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
Non-invasive Prediction of EArly Cardiac rEjectionCardiac MRI; Cell Free DNA; Heart Transplant; Pediatric Heart Transplant; RejectionRecruitingNot stated2026-08-20NCT07770659