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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
CAR-T Long Term Follow Up (LTFU) StudyLong Term Safety of Patients Receiving CAR-T in an Eligible Clinical Trial or Managed Access ProgramRecruitingPhase 32026-10-02NCT02445222
Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR GeneX-Linked Retinitis PigmentosaActive, not recruitingPhase 32026-09-25NCT04794101
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone TreatmentBone Diseases, Developmental, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Dwarfism, Musculoskeletal Abnormalities, Noonan syndrome, Skin and Connective Tissue Diseases, bone disorder, cardiovascular disorder, congenital heart disease, connective tissue disorder, endocrine system disorder, heart disorder, hereditary disease, musculoskeletal system disorderActive, not recruitingPhase 22026-09-23NCT06668805
Rapid Administration Pilot for Infusing DinutuximabNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, epithelial neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingPhase 22026-09-17NCT05421897
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
Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple SclerosisBody Weight, CNS demyelinating autoimmune disease, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Thinness, autoimmune disease, autoimmune disorder of the nervous system, demyelinating disease, immune system disorder, multiple sclerosis, nervous system disorderActive, not recruitingPhase 32026-08-10NCT01892722
A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia GravisNeoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndromeRecruitingPhase 2 / Phase 32026-07-31NCT05265273
Rethinking Early Airway Clearence TherapiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderNot yet recruitingNot applicable2026-07-10NCT07696338
Efficacy and Safety of Ofatumumab and Siponimod Compared to Fingolimod in Pediatric Patients With Multiple SclerosisCNS demyelinating autoimmune disease, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, autoimmune disease, autoimmune disorder of the nervous system, demyelinating disease, immune system disorder, multiple sclerosis, nervous system disorderActive, not recruitingPhase 32026-05-14NCT04926818
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
CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical OutcomesCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingNot stated2025-10-10NCT03350828
A Research Study to Advance the CF Therapeutics Pipeline for People Without ModulatorsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingNot stated2025-10-01NCT06504589
A Study to Evaluate the Safest Dose Range for FEIBA in Hemophilia A Patients With Inhibitors on EmicizumabHemophilia A With InhibitorActive, not recruitingPhase 42022-03-09NCT04205175