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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, pyruvate kinase deficiency of red cells, sickle cell disease, thalassemiaEnrolling by invitationPhase 42026-09-29NCT05777993
Quality of Life Before and After Transcatheter Ablation in Children With ArrythmiasArrhythmias, Cardiac, Pathologic Processes, Pathological Conditions, Signs and Symptoms, cardiovascular disorder, heart disorderRecruitingNot stated2026-09-28NCT06808204
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-ThalassemiaActive, not recruitingPhase 32026-09-28NCT04770779
Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative ColitisDigestive System Diseases, colitis, colonic disorder, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorder, ulcerative colitisActive, not recruitingPhase 32026-08-03NCT04624230
Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience.Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Psychological Trauma, Rare Diseases, Stress Disorders, Traumatic, acute stress disorder, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuria, psychiatric disorderRecruitingNot stated2026-06-24NCT06289348
Interactive Guidance Therapy With Video Feed-back of Parent and Child With Autism Spectrum Disorderautism spectrum disorder, neurodevelopmental disorder, pervasive developmental disorder, psychiatric disorderActive, not recruitingNot applicable2026-05-06NCT04607616
High-throughput Omic Technology for Identification of Biomarkers of Relapsing Acute Disseminated Encephalomyelitis in Immune Cell NetworkCNS demyelinating autoimmune disease, Chronic Disease, Disease Attributes, Leukoencephalopathies, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Post-Infectious Disorders, acute disseminated encephalomyelitis, autoimmune disease, autoimmune disorder of the nervous system, brain disorder, central nervous system disorder, demyelinating disease, immune system disorder, nervous system disorderRecruitingNot applicable2026-04-27NCT06863974
Clinico-biological Characterization and Survival of Patients With Adult T-cell Leukemia / Lymphoma (ATL) and Patients Chronically Infected With the HTLV-1 Virus (HTLV-OBS)Hemic and Lymphatic Diseases, Infections, Neoplasms by Histologic Type, RNA Virus Infections, deltaretrovirus infections, hematologic disorder, human T-lymphotropic virus 1 infectious disease, immune system disorder, immunoproliferative disorder, inborn error of immunity, leukemia, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, neoplasm, uterine carcinosarcoma, viral infectious diseaseActive, not recruitingNot stated2026-03-25NCT05237245
Respiratory and Psychological Impact of Elective Surgery of Congenital Lung MalformationsCongenital Lung MalformationsRecruitingNot stated2026-03-23NCT06424392
Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).growth delay due to insulin-like growth factor type 1 deficiencyRecruitingNot stated2026-02-06NCT00903110
Letermovir-based Dual Therapy for Treatment of Cytomegalovirus InfectionsDNA Virus Infections, Herpesviridae infectious disease, Infections, cytomegalovirus infection, viral infectious diseaseRecruitingPhase 32026-01-02NCT06334497
Understanding the Mechanisms of Autism : an MRI and Social Cognition Studyautism spectrum disorder, neurodevelopmental disorder, pervasive developmental disorder, psychiatric disorderRecruitingNot applicable2025-12-23NCT06334588
Characterization of Autoreactive Regulatory and Conventional CD4 T Cells in Recent Onset Type 1 Diabetes and Control IndividualsNutritional and Metabolic Diseases, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, immune system disorder, metabolic disease, type 1 diabetes mellitusRecruitingNot applicable2025-11-20NCT06427421
Immunomonitoring of Mold Invasive InfectionsBacterial Infections and Mycoses, Infections, Invasive Fungal Infections, fungal infectious diseaseRecruitingNot stated2025-11-20NCT06285188
Interest of Preoperative Arteriography to Identify the Adamkiewicz Artery Before Surgery for Basi-thoracic Neuroblastic TumorsNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, epithelial neoplasm, ganglioneuroblastoma, neoplasm, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumorNot yet recruitingNot stated2025-09-12NCT06798532
Safety and Efficacy Study of reSept ASD Occluder for Treating Secundum ASDCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, atrial septal defect, cardiovascular disorder, congenital heart disease, heart disorder, heart septal defectActive, not recruitingNot applicable2025-05-07NCT04591392
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ApproachCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, methylmalonic acidemia, propionic acidemiaRecruitingNot stated2024-02-05NCT04176523
Juvenile Recurrent Respiratory Papillomatosis: Establishment of a French National Cohort (PRR : National Cohort " REPA ")Juvenile Recurrent Respiratory PapillomatosisRecruitingNot stated2023-08-21NCT06000527