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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CFCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingPhase 22026-08-19NCT06237335
Study to Improve OS in 18 to 60 Year-old Patients, Comparing Daunorubicin Versus High Dose Idarubicin Induction Regimens, High Dose Versus Intermediate Dose Cytarabine Consolidation Regimens, and Standard Versus MMF Prophylaxis of GvHD in Allografted Patients in First CRHemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, hematologic disorder, leukemia, myeloid leukemia, neoplasmRecruitingPhase 2 / Phase 32026-07-17NCT02416388
A Study of CYP-001 in Combination With Corticosteroids in Adults With High-risk aGvHDacute graft versus host disease, graft versus host disease, immune system disorderActive, not recruitingPhase 22026-05-26NCT05643638
Efficacy of Intravesical Oxybutynin in Children With Neurogenic Bladder DysfunctionCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, central nervous system malformation, nervous system disorder, spina bifidaRecruitingPhase 32026-04-14NCT07027020
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
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
A Study of the Efficacy and Safety of DMX-200 in Patients With FSGS Who Are Receiving an ARBFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Urogenital Diseases, focal segmental glomerulosclerosis, glomerulonephritis, kidney disorder, nephritis, urinary system disorderRecruitingPhase 32026-02-03NCT05183646
Observatory of Patients With Haemophilia B Treated by IdElvion®Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorderActive, not recruitingNot stated2025-01-08NCT05086575