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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)Congenital, 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 32026-10-02NCT04573023
Study for Characterisation of Predictive Parameters of Clonal Evolution in Subjects With GATA2 Germline MutationHemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, bone marrow disorder, hematologic disorder, leukemia, myelodysplastic syndrome, myeloid leukemia, neoplasmRecruitingNot applicable2026-09-30NCT05983991
Pathophysiological Explorations of Red Blood Cellserythrocyte disorderRecruitingNot stated2026-09-09NCT03541525
Study of Cell-free DNA in Children and Adolescents With Acute Lymphoblastic LeukemiaHemic and Lymphatic Diseases, Neoplasm, Residual, Neoplasms by Histologic Type, Neoplastic Processes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasmRecruitingNot stated2026-08-26NCT07483476
Identification of Clinical, Genetic and Immunological Factors Involved in the Development of Severe Bacterial Infections in PediatricsBacterial Infections and Mycoses, Infections, bacterial infectious diseaseRecruitingNot applicable2026-04-03NCT07111793
Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adultschildhood malignant neoplasm, neoplasmRecruitingNot stated2026-03-31NCT04477681
Impact of First-trimester Preeclampsia Screening on Perinatal and Maternal Morbidity (RANSPRE)Female Urogenital Diseases and Pregnancy Complications, Urogenital Diseases, hypertension, pregnancy-induced, preeclampsia, pregnancy disorderRecruitingNot applicable2026-03-27NCT05521776
DCP (RaDiCo Cohort) (RaDiCo-DCP)Abnormalities, Multiple, Ciliopathies, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, hereditary disease, otorhinolaryngologic disease, primary ciliary dyskinesia, respiratory system disorderRecruitingNot stated2026-02-12NCT05951478
MPS (RaDiCo Cohort) (RaDiCo-MPS)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mucopolysaccharidosis I, Mucopolysaccharidosis III, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, Sulfatidosis, X-linked disease, brain disorder, central nervous system disorder, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, mucosulfatidosis, nervous system disorder, sphingolipidosisRecruitingNot stated2026-02-11NCT06036693
Developmental and Epileptic Encephalopathy of Genetic Etiology: Natural History Through Reuse of Clinical Datadevelopmental and epileptic encephalopathyRecruitingNot stated2025-06-29NCT06380192
High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)High-Risk Neuroblastoma; Patient With Insufficient Response ChemoimmunotherapyRecruitingPhase 32025-06-26NCT04221035