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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
SEDVasc (RaDiCo Cohort) (RaDiCo-SEDVasc)Aneurysm, Aortic Dissection, Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dissection, Blood Vessel, Ehlers-Danlos syndrome, Ehlers-Danlos syndrome, vascular type, Hemic and Lymphatic Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, cardiovascular disorder, connective tissue disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary skin disorder, skin disorder, vascular disorder, vascular hemostatic diseaseActive, not recruitingNot stated2026-02-12NCT05976841
IDMet (RaDiCo Cohort) (RaDiCo-IDMet)Abnormalities, Multiple, Angelman syndrome, Beckwith-Wiedemann syndrome, Bone Diseases, Metabolic, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Dwarfism, Imprinting Disorders, Musculoskeletal Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Overweight, Prader-Willi syndrome, Silver-Russell syndrome, Temple syndrome, bone disorder, calcium metabolic disease, central nervous system disorder, chromosomal disorder, endocrine system disorder, gonadal disorder, hereditary disease, inborn errors of metabolism, inborn metal metabolism disorder, intellectual disability, metabolic disease, movement disorder, multiple congenital anomalies due to 14q32.2 maternally expressed gene defect, musculoskeletal system disorder, nervous system disorder, nutritional disorder, obesity disorder, overnutrition, paternal uniparental disomy of chromosome 14, precocious puberty, pseudohypoparathyroidism, transient neonatal diabetes mellitusRecruitingNot stated2026-02-12NCT05945576