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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study of CD19 Targeted CAR T Cell Therapy in Pediatric Patients With Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL)B-cell neoplasm, Burkitt lymphoma, DNA Virus Infections, Disease Attributes, Epstein-Barr virus infection, Hemic and Lymphatic Diseases, Herpesviridae infectious disease, Infections, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Recurrence, Tumor Virus Infections, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, neoplasm, viral infectious diseaseRecruitingPhase 12026-10-09NCT06173518
Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 MutationsDigestive System Diseases, X-linked lymphoproliferative disease due to XIAP deficiency, digestive system disorder, enterocolitis, gastroenteritis, intestinal disorderActive, not recruitingPhase 22026-10-09NCT04641442
A Study to Evaluate the Safety and Efficacy of JZP926 Capsule for the Treatment of Juvenile Myoclonic EpilepsyEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, juvenile myoclonic epilepsy, nervous system disorderRecruitingPhase 2 / Phase 32026-10-06NCT07723963
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 to Evaluate the Efficacy and Safety of Cannabidiol Oral Solution (CBD-OS [GWP42003-P, JZP926]) for the Treatment of Focal-Onset SeizuresNeurologic Manifestations, Pathological Conditions, Signs and Symptoms, Seizures, Focal, Signs and Symptoms, nervous system disorder, visual epilepsyRecruitingPhase 12026-10-02NCT07233239
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
Long-term Safety Study of Rimegepant in Pediatric Subjects for the Acute Treatment of MigraineEar 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 32026-09-24NCT04743141
ACT001 for the Treatment of Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade GliomasNeoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, diffuse intrinsic pontine glioma, epithelial neoplasm, glioma, neoplasm, nervous system disorder, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingPhase 22026-09-21NCT06838676
Treatment of Obstructive Sleep Apnea With Personalized Surgery in Children With Down Syndrome (TOPS-DS)Abnormalities, Multiple, Apnea, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Down syndrome, Dyssomnias, Neurobehavioral Manifestations, Neurologic Manifestations, Respiration Disorders, Sleep Disorders, Intrinsic, chromosomal disorder, hereditary disease, intellectual disability, nervous system disorder, obstructive sleep apnea syndrome, respiratory system disorder, sleep apnea syndrome, sleep-wake disorderRecruitingNot applicable2026-09-11NCT05508971
Safety and Efficacy Study of Cenobamate in Pediatric Subjects 2-17 Years of Age With Partial-onset (Focal) SeizuresNeurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, focal epilepsy, nervous system disorder, visual epilepsyActive, not recruitingPhase 32026-09-09NCT05067634
Pediatric Influence of Cooling Duration on Efficacy in Cardiac Arrest Patients (P-ICECAP)Body Temperature Changes, Coma, Consciousness Disorders, Hypothermia, Hypoxia, Hypoxia, Brain, Neurobehavioral Manifestations, Neurologic Manifestations, Out-of-Hospital Cardiac Arrest, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Signs and Symptoms, Respiratory, Unconsciousness, brain disorder, brain hypoxia - ischemia, brain ischemia, cardiac arrest, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, heart disorder, nervous system disorder, vascular disorderRecruitingNot applicable2026-09-08NCT05376267
Assessment of Worldwide Acute Kidney Injury Epidemiology in Neonates 2.0Chronic Disease, Disease Attributes, Edema, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Obstetric Labor Complications, Obstetric Labor, Premature, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Premature Birth, Signs and Symptoms, Urogenital Diseases, acute kidney injury, cardiovascular disorder, chronic renal failure syndrome, hypertensive disorder, kidney disorder, kidney failure, pregnancy disorder, urinary system disorder, vascular disorderEnrolling by invitationNot stated2026-09-04NCT06823115
Clinical Course Of Disease In Participants With FA-CMCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebellar disorder, heart disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorderRecruitingNot stated2026-09-02NCT06865482
X-linked Hypophosphatemia Disease Monitoring ProgramBone Diseases, Metabolic, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hypophosphatemia, Familial, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Rickets, X-Linked Hypophosphatemic, Urogenital Diseases, X-linked hypophosphatemic rickets, bone disorder, calcium metabolic disease, hereditary disease, hypophosphatemia, hypophosphatemic rickets, inborn errors of metabolism, inborn metal metabolism disorder, kidney disorder, metabolic disease, musculoskeletal system disorder, nutritional deficiency disease, nutritional disorder, phosphorus metabolism disease, renal tubular transport disease, rickets, urinary system disorder, vitamin D deficiency, vitamin deficiency disorderActive, not recruitingNot stated2026-08-28NCT03651505
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
Observational Study of THC Concentrations in Acute Cannabis-induced CNS DepressionAcute Cannabis-induced CNS DepressionRecruitingNot stated2026-07-30NCT06017622
Comparative Effectiveness of Available Treatments for Sinus and Ear Infections in Children (Acute Sinusitis)Infections, nasal disorder, otorhinolaryngologic disease, paranasal sinus disorder, respiratory system disorder, respiratory tract infectious disorder, sinusitisNot yet recruitingPhase 42026-07-29NCT07732699
Comparative Effectiveness of Available Treatments for Sinus and Ear Infections in Children (Acute Otitis Media)Ear Diseases, otitis media, otorhinolaryngologic diseaseNot yet recruitingPhase 42026-07-28NCT07730814
Phase 2b Study of RPT904 as Monotherapy in Participants With IgE-Mediated Food AllergyEgg Hypersensitivity, Hypersensitivity, Immediate, Milk Hypersensitivity, Nut and Peanut Hypersensitivity, Peanut Hypersensitivity, food allergy, immune system disorderRecruitingPhase 22026-07-28NCT07220811
COMPASSION S3 - Evaluation of the SAPIEN 3 Transcatheter Heart Valve in Patients With Pulmonary Valve DysfunctionCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Pulmonary Atresia, Ventricular Outflow Obstruction, aortic valve disorder, aortopulmonary window, cardiovascular disorder, congenital heart disease, heart disorder, heart septal defect, heart valve disorder, persistent truncus arteriosus, pulmonary valve insufficiency, pulmonary valve stenosis, tetralogy of fallot, transposition of the great arteries, vascular malformationRecruitingNot applicable2026-07-23NCT02744677
Health-E You Efficacy Trial for Male AdolescentsDisease Attributes, Genital Diseases, Infections, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, infectious disease, sexually transmitted diseaseRecruitingNot applicable2026-07-15NCT06525064
Adalimumab Dose Adjustment for Pediatric Uveitis Treatment Trialanterior uveitis, eye disorder, intermediate uveitis, panuveitis, uveal disorder, uveitisNot yet recruitingPhase 42026-07-13NCT07699107
Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- InfantCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Sex Chromosome Disorders, X-linked disease, chromosomal disorder, fragile X syndrome, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorderRecruitingNot applicable2026-07-10NCT06560242
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)Bone Marrow Failure Disorders, Chronic Disease, Disease Attributes, Hemic and Lymphatic Diseases, Hodgkins lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myeloproliferative Disorders, Neoplasms by Histologic Type, Neoplasms by Site, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, acute lymphoblastic leukemia, acute myeloid leukemia, anemia, aplastic anemia, autoimmune disease, bone marrow disorder, chronic myeloid leukemia, disease, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, immunoproliferative disorder, inborn errors of metabolism, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, myeloid leukemia, neoplasm, non-Hodgkin lymphomaRecruitingNot stated2026-07-06NCT01351545