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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Pharmacokinetics, Safety, and Efficacy of Povorcitinib in Adolescent Participants With Moderate to Severe Hidradenitis SuppurativaBacterial Infections and Mycoses, Infections, Skin and Connective Tissue Diseases, Suppuration, bacterial infectious disease, hidradenitis, hidradenitis suppurativa, skin disease caused by bacterial infection, skin disorder, skin disorder caused by infection, sweat gland disorderRecruitingPhase 22026-10-09NCT07213973
A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, von Willebrand disease (hereditary or acquired), von Willebrand disease 3RecruitingPhase 32026-10-09NCT06998524
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
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Obinutuzumab in Adolescents With Active Class III or IV Lupus Nephritis and the Safety and PK of Obinutuzumab in Pediatric ParticipantsFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Skin and Connective Tissue Diseases, Urogenital Diseases, autoimmune disease, connective tissue disorder, glomerulonephritis, immune system disorder, kidney disorder, lupus nephritis, nephritis, systemic lupus erythematosus, urinary system disorderRecruitingPhase 22026-10-06NCT05039619
Neonatal Enhanced Recovery After Surgery (ERAS) Outcomes StudyCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and AbnormalitiesNot yet recruitingNot stated2026-10-01NCT07771478
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
8 vs 12 Weeks: A Pavlik Harness TrialCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hip Dislocation, Hip Dislocation, Congenital, Hip Dysplasia, Congenital, Nonsyndromic, Hip Injuries, Joint Dislocations, Musculoskeletal Abnormalities, arthropathy, developmental dysplasia of the hip, injury, musculoskeletal system disorderNot yet recruitingNot applicable2026-09-29NCT07847619
A Study to Find Out How Nerandomilast is Tolerated, Handled by the Body, and if it Helps Children and Adolescents With Interstitial Lung Disease (FIBRONEER-chILD)Fibrosing Interstitial Lung DiseaseRecruitingPhase 32026-09-29NCT07366034
HCRN Endoscopic Versus Shunt Treatment of Hydrocephalus in Infantsbrain disorder, central nervous system disorder, hydrocephalus, nervous system disorderActive, not recruitingNot applicable2026-09-25NCT04177914
Effect of Metformin on Behaviour and the Brain in Children Treated for a Brain TumourCognition Disorders, Cognitive Dysfunction, Medulloblastoma, Childhood, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, cognitive disorder, epithelial neoplasm, glioma, medulloblastoma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumor, psychiatric disorderRecruitingPhase 32026-09-24NCT05230758
Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment OptionsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Ewing sarcoma, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Head and Neck Neoplasms, Male Urogenital Diseases, Mouth Neoplasms, Neoplasm Metastasis, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neoplastic Processes, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, Stomatognathic Diseases, Thyroid Cancer, Papillary, Urogenital Diseases, Urogenital Neoplasms, Wilms tumor, adenocarcinoma, benign urinary system neoplasm, brain disorder, carcinoma, central nervous system cancer, central nervous system disorder, connective tissue neoplasm, endocrine system disorder, epithelial neoplasm, glioma, hereditary disease, hereditary neoplastic syndrome, kidney cancer, kidney disorder, malignant endocrine neoplasm, medulloblastoma, mesoblastic nephroma, mixed neoplasm, mouth disorder, neoplasm, nervous system disorder, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, papillary adenocarcinoma, primitive neuroectodermal tumor, salivary gland cancer, salivary gland disorder, sarcoma, thyroid gland disorder, thyroid tumor, urinary system disorderActive, not recruitingPhase 1 / Phase 22026-09-23NCT02650401
Efficacy and Safety of Erenumab in Pediatric Participants With Episodic MigraineHeadache, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, headache disorder, hypnic headache, migraine disorder, nervous system disorderActive, not recruitingPhase 32026-09-21NCT03836040
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or ThalassaemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease, thalassemiaRecruitingPhase 32026-09-18NCT06609226
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients 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 22026-09-18NCT06198712
A Study to Find Out How EMPAgliflozin is Tolerated and if it Helps Children and Adolescents With Chronic KIDNEY Disease (EMPA-KIDNEY® Kids)Chronic 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 32026-09-16NCT07107945
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
A Study to Investigate the Transition of Children From 'Artisanal" Cannabidiol (CBD) to EpidiolexCongenital, 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, epilepsy, hereditary disease, nervous system disorder, visual epilepsyNot yet recruitingPhase 42026-09-11NCT06924827
A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia 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 disorderActive, not recruitingPhase 32026-09-09NCT05685238
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) DeficiencyBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, ornithine carbamoyltransferase deficiency, urea cycle disorderActive, not recruitingPhase 32026-09-09NCT05345171
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 2 / Phase 32026-09-09NCT05115110
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Dyskinesias, Friedreich ataxia, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Signs and Symptoms, Spinocerebellar Degenerations, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorderRecruitingNot stated2026-09-01NCT06016946
A Study to Evaluate the Drug Levels, Efficacy and Safety of Deucravacitinib in Children and Adolescent Participants With Moderate to Severe Plaque PsoriasisSkin Diseases, Papulosquamous, Skin and Connective Tissue Diseases, psoriasis, skin disorderRecruitingPhase 32026-08-18NCT04772079
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS TumorsNeoplasms by Site, central nervous system cancer, neoplasm, nervous system disorderActive, not recruitingPhase 1 / Phase 22026-08-14NCT04774718
An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care TreatmentCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, von Willebrand disease (hereditary or acquired), von Willebrand disease 3Active, not recruitingNot stated2026-08-10NCT06883240
Anesthetics and Analgesics in ChildrenNeurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and SymptomsRecruitingNot stated2026-08-05NCT03427736