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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and SarcomasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Ewing sarcoma, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Hodgkins lymphoma, 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, astrocytoma (excluding glioblastoma), benign urinary system neoplasm, brain disorder, brainstem cancer, central nervous system cancer, central nervous system disorder, connective tissue neoplasm, desmoplastic small round cell tumor, diffuse intrinsic pontine glioma, ependymoma, epithelial neoplasm, glioblastoma, glioma, gliosarcoma, hereditary disease, hereditary neoplastic syndrome, immune system disorder, immunoproliferative disorder, kidney cancer, kidney disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, medulloblastoma, mixed neoplasm, myomatous neoplasm, neoplasm, nervous system disorder, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, pediatric lymphoma, primitive neuroectodermal tumor, rhabdoid tumor, rhabdomyosarcoma, sarcoma, synovial sarcoma, urinary system disorderActive, not recruitingPhase 12026-10-01NCT03478462
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Infant Death, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderActive, not recruitingPhase 2 / Phase 32026-09-09NCT05371613
Janus Kinase (JAK) Inhibitors to Preserve C-Peptide Production in New Onset Type 1 Diabetes (T1D)Nutritional and Metabolic Diseases, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, immune system disorder, metabolic disease, type 1 diabetes mellitusActive, not recruitingPhase 22026-09-02NCT05743244
Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive ImpairmentCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderActive, not recruitingPhase 2 / Phase 32025-11-06NCT06031259