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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
Long-Term Extension Study of Vosoritide to Treat Children With HypochondroplasiahypochondroplasiaEnrolling by invitationPhase 32026-09-09NCT07073014
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingPhase 2 / Phase 32026-08-10NCT06328608
Interventional Study of Vosoritide for the Treatment of Children With HypochondroplasiaBone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, bone disorder, endocrine system disorder, hereditary disease, hypochondroplasia, musculoskeletal system disorder, osteochondrodysplasiaActive, not recruitingPhase 32026-01-20NCT06455059
The U.K. NorthStar Clinical NetworkCongenital, 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 diseaseNot yet recruitingNot stated2024-12-02NCT06711692
Paediatric Sjögren Syndrome Cohort Study and Repository (PaedSSCoRe)Sjogren's Syndrome Childhood-onsetActive, not recruitingNot stated2021-04-19NCT04762108