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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell TransplantationCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseActive, not recruitingPhase 22026-09-24NCT04018937
Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN)Bone Diseases, Developmental, Collagen Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia, osteogenesis imperfectaRecruitingPhase 22026-08-17NCT07062588
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingPhase 2 / Phase 32026-08-10NCT06328608
Infant Formula in Infants and Children With Cow's Milk AllergyCow's Milk Allergy, Hypersensitivity, Immediate, Milk Hypersensitivity, food allergy, immune system disorderRecruitingNot applicable2026-07-15NCT06456541
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 32026-06-01NCT05337553
Point of Care Lung Ultrasound in Preterm Infants With Respiratory DistressCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Infant, Newborn, Diseases, Infant, Premature, Diseases, Lung Injury, Ventilator-Induced Lung Injury, bronchopulmonary dysplasia, lung disorder, respiratory system disorderEnrolling by invitationNot stated2026-05-22NCT05925075
Single Arm Romiplostim to Prevent CITEwing sarcoma, Neoplasms by Histologic Type, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, connective tissue neoplasm, neoplasm, osteosarcoma, sarcomaRecruitingEarly phase 12026-03-18NCT07048249
A Clinical Trial of Soluble Fiber for AsthmaDisorders of Environmental Origin, Environmental Illness, Hypersensitivity, Immediate, Respiratory Hypersensitivity, asthma, bronchial disorder, immune system disorder, lung disorder, obstructive lung disease, respiratory system disorderRecruitingPhase 22026-03-04NCT06372249
Project Mountain - Comparing SpO2 and SaO2 for Accuracynasal alar collapse, bilateralRecruitingNot stated2025-04-02NCT06372106
InfasurfAero™ Versus Sham Treatment in Preterm Newborns With RDSDeath, Female Urogenital Diseases and Pregnancy Complications, Obstetric Labor Complications, Obstetric Labor, Premature, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Perinatal Death, Premature Birth, Pulmonary Atelectasis, Respiration Disorders, Respiratory Aspiration, Urogenital Diseases, adult acute respiratory distress syndrome, lung disorder, pregnancy disorder, respiratory system disorderRecruitingPhase 32025-03-30NCT05960929