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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host DiseaseBronchiolitis Obliterans, bronchial disorder, bronchiolitis, bronchiolitis obliterans syndrome, bronchitis, cryptogenic organizing pneumonia, graft versus host disease, immune system disorder, lung disorder, obstructive lung disease, respiratory system disorderRecruitingPhase 32026-10-09NCT06585774
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Sickle Cell Trait, Vaso-Occlusive Crises, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseRecruitingPhase 32026-09-28NCT06439082
Pediatric Long-Term Follow-up and Rollover StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Oligodendroglioma, Childhood, anaplastic astrocytoma, anaplastic ganglioglioma, anaplastic oligodendroglioma, anaplastic pleomorphic xanthoastrocytoma, angiocentric glioma, astrocytoma (excluding glioblastoma), brain disorder, central nervous system cancer, central nervous system disorder, central neurocytoma, cerebellar liponeurocytoma, chordoid glioma of the third ventricle, diffuse astrocytoma, epithelial neoplasm, extraventricular neurocytoma, gangliocytoma, ganglioglioma, glioblastoma, glioma, hereditary disease, hereditary neoplastic syndrome, histiocytosis, inherited neurodegenerative disorder, interstitial lung disease, lung disorder, lymphatic system disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neuroblastoma, neurocutaneous syndrome, neurodegenerative disease, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, oligodendroglioma, papillary glioneuronal tumor, peripheral nervous system disorder, peripheral nervous system neoplasm, pilocytic astrocytoma, plexiform neurofibroma, primitive neuroectodermal tumor, respiratory system disorder, rosette-forming glioneuronal tumor of fourth ventriculeActive, not recruitingPhase 42026-08-27NCT03975829
Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive PatientsB-cell acute lymphoblastic leukemia, B-cell neoplasm, Burkitt lymphoma, DNA Virus Infections, Epstein-Barr virus infection, Hemic and Lymphatic Diseases, Herpesviridae infectious disease, Infections, Neoplasm, Residual, Neoplasms by Histologic Type, Neoplastic Processes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Tumor Virus Infections, immune system disorder, immunoproliferative disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, neoplasm, viral infectious diseaseActive, not recruitingPhase 22026-08-19NCT03876769
Selective Antigen Specific T Cells and CAR T Cells in Subjects With Relapsed/Refractory Embryonal Tumors (SABRE)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ewing sarcoma, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Myosarcoma, 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, Urogenital Diseases, Urogenital Neoplasms, Wilms tumor, benign urinary system neoplasm, connective tissue neoplasm, epithelial neoplasm, hereditary disease, hereditary neoplastic syndrome, kidney cancer, kidney disorder, mixed neoplasm, myomatous neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, primitive neuroectodermal tumor, rhabdomyosarcoma, sarcoma, urinary system disorderRecruitingPhase 12026-08-13NCT07172958
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial HypercholesterolemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Ventricular Outflow Obstruction, aortic valve disorder, aortic valve stenosis, cardiovascular disorder, familial hypercholesterolemia, heart disorder, heart failure, heart valve disorder, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 32026-07-24NCT06597006
A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's DisorderCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Tourette syndrome, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, neurodevelopmental disorder, psychiatric disorder, tic disorderActive, not recruitingPhase 32026-07-22NCT06021522
Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Vaso-Occlusive Crises, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseRecruitingPhase 42026-06-08NCT04657822
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial HypercholesterolemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Ventricular Outflow Obstruction, aortic valve disorder, aortic valve stenosis, cardiovascular disorder, familial hypercholesterolemia, heart disorder, heart failure, heart valve disorder, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 32026-05-15NCT06597019
A Study to Learn About The COVID-19 (Study) Vaccine (Called COMIRNATY) in People That Are Less Than 21 Years Old.COVID-19, Coronaviridae Infections, Infections, Nidovirales infectious disease, Orthocoronavirinae infectious disease, RNA Virus Infections, cardiomyopathy, cardiovascular disorder, heart disorder, lung disorder, myocarditis, pneumonia, respiratory system disorder, respiratory tract infectious disorder, viral infectious disease, viral pneumoniaActive, not recruitingPhase 42026-05-07NCT05295290