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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Phase 2 Study to Assess the Safety and Efficacy of ANG003Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, exocrine pancreatic insufficiency, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingPhase 22026-10-06NCT07450547
Bowel Continence Across the Lifespan in People With Spina BifidaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Fecal Incontinence, central nervous system malformation, colonic disorder, digestive system disorder, functional colonic disease, intestinal disorder, nervous system disorder, neurogenic bowel, rectal disorder, spina bifidaRecruitingNot stated2026-09-24NCT07390318
Liver Transplantation From Donors With HIV: Impact on Opportunistic Infections, Cancer, and Long-Term Outcomes (Expanding HOPE Liver)HIVRecruitingNot applicable2026-09-09NCT07665372
Coupled Gentamicin-Lactobacillus Rhamnosus in NLUTDCNS demyelinating autoimmune disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Infections, Male Urogenital Diseases, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Urinary Bladder, Neurogenic, Urogenital Diseases, autoimmune disease, autoimmune disorder of the nervous system, central nervous system disorder, central nervous system malformation, demyelinating disease, immune system disorder, injury, multiple sclerosis, nervous system disorder, nervous system injury, spina bifida, spinal cord disorder, spinal cord injury, urinary bladder disorder, urinary system disorder, urinary tract infectionNot yet recruitingEarly phase 12026-08-21NCT07643974
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuriaRecruitingPhase 1 / Phase 22026-08-18NCT06332807
A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia GravisNeoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndromeRecruitingPhase 2 / Phase 32026-07-31NCT05265273
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With PhenylketonuriaBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuriaActive, not recruitingPhase 32026-07-21NCT05270837
Study to Enable New Diagnostics for Pulmonary Microbes in People With CFCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingNot stated2026-07-02NCT07312734
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuriaRecruitingNot stated2026-03-25NCT05813678
Strength and Muscle Related Outcomes for Nutrition and Lung Function in 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 recruitingNot stated2026-03-13NCT05639556
Effect of Raised End-Tidal pCo2 on Choroidal VolumeIntraocular PressureRecruitingNot applicable2026-01-09NCT06275308
Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingNot stated2025-11-03NCT06683092
Prospective Study of Pregnancy in Women With Cystic FibrosisCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingNot stated2025-11-03NCT04828382
A Research Study to Advance the CF Therapeutics Pipeline for People Without ModulatorsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingNot stated2025-10-01NCT06504589
IV Gallium Study for Patients With Cystic Fibrosis Who Have NTM (ABATE Study)Actinomycetales infectious disease, Bacterial Infections and Mycoses, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, Infections, Mycobacterium Infections, Nontuberculous, Mycobacterium avium complex disease, bacterial infectious disease, cystic fibrosis, gram-positive bacterial infections, hereditary disease, lung disorder, mycobacterial infectious disease, pancreas disorder, respiratory system disorderRecruitingPhase 12025-10-01NCT04294043
Strategy to Avoid Excessive Oxygen in Major Burn PatientsCritical Illness, Disease Attributes, Hyperoxia, Hypoxia, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Signs and Symptoms, Respiratory, burn, injuryActive, not recruitingPhase 32025-04-02NCT04534972
Strategy to Avoid Excessive Oxygen for Critically Ill Trauma PatientsCritical Illness, Disease Attributes, Hyperoxia, Hypoxia, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Signs and Symptoms, Respiratory, burn, injuryActive, not recruitingPhase 32025-04-02NCT04534959
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2025-01-31NCT04174157