23 results · Page 1 of 1

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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Evaluate the Efficacy and Safety of Ruxolitinib Cream in Children (6 to < 12 Years Old) With Nonsegmental VitiligoNonSegmental VitiligoRecruitingPhase 32026-10-09NCT06804811
A Study of Mirikizumab (LY3074828) in Pediatric Participants With Crohn's DiseaseCrohn disease, Digestive System Diseases, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorderRecruitingPhase 32026-10-07NCT05509777
A Study of Baricitinib in Participants From 1 Year to Less Than 18 Years Old With Juvenile Idiopathic ArthritisSkin and Connective Tissue Diseases, arthritic joint disease, arthropathy, autoimmune disease, connective tissue disorder, immune system disorder, juvenile idiopathic arthritis, musculoskeletal system disorder, rheumatic disorderRecruitingPhase 32026-10-07NCT03773965
A Study of Repotrectinib in Pediatric and Young Adult Subjects Harboring ALK, ROS1, OR NTRK1-3 AlterationsGranuloma, Hemic and Lymphatic Diseases, Neoplasm Metastasis, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neoplastic Processes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, T-cell non-Hodgkin lymphoma, anaplastic large cell lymphoma, astrocytoma (excluding glioblastoma), connective tissue neoplasm, epithelial neoplasm, glioblastoma, glioma, immune system disorder, immunoproliferative disorder, inflammatory myofibroblastic tumor, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumor, sarcoma, solitary fibrous tumorRecruitingPhase 1 / Phase 22026-09-25NCT04094610
Randomized Study in Children and Adolescents With Migraine: Acute TreatmentEar Diseases, Nausea, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Photophobia, Sensation Disorders, Signs and Symptoms, Signs and Symptoms, Digestive, brain disorder, central nervous system disorder, eye disorder, headache disorder, hearing disorder, hyperacusis, hypnic headache, migraine disorder, nervous system disorder, otorhinolaryngologic disease, vision disorderRecruitingPhase 32026-09-24NCT04649242
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)Epilepsies, 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, neurodevelopmental disorder, psychiatric disorder, visual epilepsyActive, not recruitingPhase 32026-09-17NCT06660394
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Lennox-Gastaut syndrome, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, developmental and epileptic encephalopathy, epilepsy, hereditary disease, nervous system disorder, neurodevelopmental disorder, psychiatric disorder, visual epilepsyEnrolling by invitationPhase 32026-09-15NCT06908226
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathies (DEE)Congenital, 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, developmental and epileptic encephalopathy, epilepsy, hereditary disease, nervous system disorder, neurodevelopmental disorder, psychiatric disorder, visual epilepsyActive, not recruitingPhase 32026-09-15NCT06719141
A Study of Eptinezumab in Pediatric Participants With Episodic MigraineEpisodic MigraineRecruitingPhase 32026-09-09NCT05897320
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 Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA VariantsBrain 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 32026-09-01NCT06904261
A Study With Eptinezumab in Adolescents (12-17 Years) With Chronic MigraineChronic Migraine in ChildrenRecruitingPhase 32026-08-21NCT04965675
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)Alexander disease, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, demyelinating disease, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative diseaseActive, not recruitingPhase 32026-08-21NCT04849741
Imlifidase Prior to Kidney Transplant in Highly Sensitised ChildrenChronic 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 22026-08-18NCT05753930
A Study of Baricitinib (LY3009104) in Participants From 1 Year to Less Than 18 Years Old With Systemic Juvenile Idiopathic Arthritis (sJIA)Body Temperature Changes, Fever, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Skin and Connective Tissue Diseases, arthritic joint disease, arthropathy, autoimmune disease, connective tissue disorder, immune system disorder, juvenile idiopathic arthritis, musculoskeletal system disorder, rheumatic disorderActive, not recruitingPhase 32026-08-11NCT04088396
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin MutationsDeafness, Autosomal Recessive 9, Ear Diseases, Hearing Loss, Sensorineural, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, auditory neuropathy, hearing disorder, hearing loss disorder, nervous system disorder, otorhinolaryngologic diseaseRecruitingPhase 1 / Phase 22026-08-05NCT05788536
Multi-omics Study in Citrin DeficiencyAdult-onset citrullinemia type 2Active, not recruitingNot stated2026-06-02NCT06895746
A Study of Baricitinib (LY3009104) in Participants From 2 Years to Less Than 18 Years Old With Active JIA-Associated Uveitis or Chronic Anterior Antinuclear Antibody-Positive Uveitiseye disorder, uveal disorder, uveitisActive, not recruitingPhase 32026-04-24NCT04088409
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys 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 42026-04-23NCT06713135
A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 StudyAcid-Base Imbalance, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, acidosis disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, propionic acidemiaRecruitingPhase 1 / Phase 22026-03-23NCT05130437
Open-Label Study of mRNA-3927 in Participants With Propionic AcidemiaAcid-Base Imbalance, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, acidosis disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, propionic acidemiaRecruitingPhase 1 / Phase 22026-01-22NCT04159103
Muscle Connective Tissue in Limb Development and DiseaseRadial DysplasiaRecruitingNot stated2025-08-27NCT02611089
Honeycomb: Evaluation of Radiprodil in Children with GRIN-related DisorderGRIN-related DisordersActive, not recruitingPhase 12024-11-15NCT05818943