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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseRecruitingPhase 22026-10-01NCT06578507
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's AtaxiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorderActive, not recruitingNot stated2026-07-21NCT05874388
Fluid-responsiveness in Children Ventilated With LOW Tidal Volume - The FLOW Study.Pathologic Processes, Pathological Conditions, Signs and Symptoms, ShockRecruitingNot applicable2026-04-07NCT07494643
FARD (RaDiCo Cohort) (RaDiCo-FARD)Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Conjunctival Diseases, Eye Diseases, Hereditary, Infant, Newborn, Diseases, Keratoderma, Palmoplantar, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, Nutritional and Metabolic Diseases, Pemphigoid, Benign Mucous Membrane, Skin Abnormalities, Skin and Connective Tissue Diseases, albinism, autoimmune disease, ectodermal dysplasia syndrome, eye disorder, hereditary disease, hereditary neoplastic syndrome, hereditary skin disorder, hypopigmentation of the skin, ichthyosis, immune system disorder, inborn disorder of amino acid metabolism, inborn errors of metabolism, incontinentia pigmenti, inherited epidermolysis bullosa, inherited neurodegenerative disorder, keratosis, metabolic disease, mucous membrane pemphigoid, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, pemphigus, peripheral nervous system disorder, skin disorder, skin pigmentation disorder, vesiculobullous skin diseaseRecruitingNot stated2026-02-12NCT05954416
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
A Multicenter Multinational Observational Study of Children With HypochondroplasiahypochondroplasiaRecruitingNot stated2025-04-03NCT06212947
EuroSIDA - Clinical and Virological Outcome of European Patients Infected With HIVAIDS, Acid-Base Imbalance, Blood-Borne Infections, Chronic Disease, DNA Virus Infections, Digestive System Diseases, Disease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Flaviviridae infectious disease, Genital Diseases, HIV infectious disease, Hepadnaviridae infectious disease, Hepatic Insufficiency, Infections, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, RNA Virus Infections, Signs and Symptoms, Slow Virus Diseases, Urination Disorders, Urogenital Diseases, Urological Manifestations, acidosis disorder, bone fracture, cardiovascular disorder, chronic liver failure, chronic renal failure syndrome, coinfection, diabetes mellitus, endocrine system disorder, glucose metabolism disease, hepatitis, hepatitis B virus infection, hepatitis C virus infection, immune system disorder, inborn error of immunity, infectious disease, injury, kidney disorder, kidney failure, lactic acidosis, lentivirus infection, liver disorder, liver failure, metabolic disease, proteinuria, sexually transmitted disease, urinary system disorder, uterine carcinosarcoma, viral hepatitis, viral infectious disease, viral sexually transmitted diseaseEnrolling by invitationNot stated2024-04-12NCT02699736