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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia ACongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorderRecruitingPhase 1 / Phase 22026-10-08NCT05987449
A Study of Ifinatamab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01D/LIGHTBEAM-U01)cancer, neoplasmRecruitingPhase 1 / Phase 22026-10-06NCT07630974
A Study of Patritumab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01C/LIGHTBEAM-U01)cancer, neoplasmRecruitingPhase 1 / Phase 22026-10-06NCT06941272
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without InhibitorsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, severe hemophilia AActive, not recruitingPhase 32026-10-01NCT04431726
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 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
A Study to Learn More About How Safe the Study Treatment Finerenone is in Long-term Use When Taken With an ACE Inhibitor or Angiotensin Receptor Blocker Over 18 Months of Use in Children and Young Adults From 1 to 18 Years of Age With Chronic Kidney Disease and ProteinuriaChronic Disease, Disease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Urination Disorders, Urogenital Diseases, Urological Manifestations, chronic renal failure syndrome, kidney disorder, kidney failure, proteinuria, urinary system disorderRecruitingPhase 32026-09-24NCT05457283
A Study to Evaluate the Safety, Tolerability, and Efficacy of BIIB017 (Peginterferon Beta-1a) in Pediatric Participants for the Treatment of Relapsing-Remitting Multiple SclerosisCNS demyelinating autoimmune disease, autoimmune disease, autoimmune disorder of the nervous system, demyelinating disease, immune system disorder, multiple sclerosis, nervous system disorder, relapsing-remitting multiple sclerosisActive, not recruitingPhase 32026-09-24NCT03958877
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
Study of Oral Upadacitinib and Subcutaneous/Intravenous Tocilizumab to Evaluate Change in Disease Activity, Adverse Events and How Drug Moves Through the Body of Pediatric and Adolescent Participants With Active Systemic Juvenile Idiopathic Arthritis.Skin and Connective Tissue Diseases, arthritic joint disease, arthropathy, autoimmune disease, connective tissue disorder, immune system disorder, juvenile idiopathic arthritis, musculoskeletal system disorder, rheumatic disorderRecruitingPhase 32026-09-15NCT05609630
HENKA: Growing in Emotional Well-beingEmotional Well-being; Mental Health; Resilience, PsychologicalActive, not recruitingNot applicable2026-09-14NCT07796867
Study of the Adverse Events and Change in Disease State of Pediatric Participants (and Young Adults Between the Ages of 18-25) With Relapsed/Refractory Aggressive Mature B-cell Neoplasms Receiving Subcutaneous (SC) Injections of EpcoritamabB-cell neoplasm, Disease Attributes, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, diffuse large B-cell lymphoma, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, neoplasm, non-Hodgkin lymphomaActive, not recruitingPhase 12026-09-11NCT05206357
A Study to Assess Adverse Events, Change in Disease Activity, and How the Drug Moves Through the Body in Children With Juvenile Psoriatic Arthritis (jPsA) Receiving Subcutaneously Injected Risankizumab or AdalimumabSkin Diseases, Papulosquamous, Skin and Connective Tissue Diseases, Spondylarthritis, Spondylarthropathies, arthritic joint disease, arthropathy, autoimmune disease, bone disorder, connective tissue disorder, immune system disorder, juvenile idiopathic arthritis, musculoskeletal system disorder, psoriasis, psoriatic arthritis, rheumatic disorder, skin disorder, spondylitis, vertebral column disorderRecruitingPhase 32026-09-01NCT06100744
Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Left Ventricular Systolic Dysfunction (MK-1242-036)Left Ventricular Systolic Dysfunction, Ventricular Dysfunction, Ventricular Dysfunction, Left, cardiovascular disorder, heart disorder, heart failureRecruitingPhase 2 / Phase 32026-08-28NCT05714085
A Study to Learn More About How Well the Study Treatment Finerenone Works, How Safe it is, How it Moves Into, Through, and Out of the Body, and the Effects it Has on the Body When Taken With an ACE Inhibitor or Angiotensin Receptor Blocker in Children With Chronic Kidney Disease and ProteinuriaChronic Disease, Disease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Urination Disorders, Urogenital Diseases, Urological Manifestations, chronic renal failure syndrome, kidney disorder, kidney failure, proteinuria, urinary system disorderActive, not recruitingPhase 32026-08-28NCT05196035
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
A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)Abnormalities, Multiple, Ciliopathies, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Polycystic Kidney, Autosomal Recessive, Urogenital Diseases, cystic kidney disease, hereditary disease, kidney disorder, oligohydramnios, polycystic kidney disease, pregnancy disorder, urinary system disorderRecruitingPhase 32026-08-07NCT04782258
A Study to Assess Adverse Events and Change in Disease Activity Comparing Oral Upadacitinib to Subcutaneous Dupilumab in Children From 2 to Less Than 12 Years of Age With Moderate to Severe Atopic DermatitisCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hypersensitivity, Immediate, Skin Diseases, Eczematous, Skin and Connective Tissue Diseases, atopic eczema, dermatitis, hereditary disease, hereditary skin disorder, immune system disorder, skin disorderRecruitingPhase 32026-08-06NCT06461897
A Study to Learn About the Safety of Vedolizumab and How Well it Works in Children and Teenagers With Active Chronic PouchitisCrohn ileitis, Digestive System Diseases, Ileal Diseases, digestive system disorder, enteritis, gastroenteritis, intestinal disorder, pouchitisRecruitingPhase 32026-08-03NCT06443502
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Upadacitinib in Pediatric Subjects With Polyarticular Course 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 disorderActive, not recruitingPhase 12026-08-03NCT03725007
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of 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 32026-07-22NCT04281485
Beeline: A Phase 3 Study in GRIN-related Neurodevelopmental DisorderGRIN-related complex neurodevelopmental disorderRecruitingPhase 32026-07-17NCT07224581
A Phase 2 Study of CRD-4730 in CPVTArrhythmias, Cardiac, Cardiac Conduction System Disease, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Polymorphic Catecholaminergic Ventricular Tachycardia, Tachycardia, cardiovascular disorder, heart disorder, ventricular tachycardiaRecruitingPhase 22026-07-09NCT06658899