Great Ormond Street Hospital

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Trial	Conditions	Status	Phase	Updated	NCT ID
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome	Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, generalized epilepsy with febrile seizures plus, type 2, nervous system disorder	Recruiting	Phase 1 / Phase 2	2026-09-22	NCT05419492
Pediatric Patients Aged 1 to 6 Years With APDS	activated PI3K-delta syndrome	Active, not recruiting	Phase 3	2026-06-15	NCT05693129
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome	Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, nervous system disorder	Active, not recruiting	Phase 1 / Phase 2	2026-06-08	NCT06283212
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, methylmalonic acidemia, propionic acidemia	Recruiting	Not stated	2024-02-05	NCT04176523

Clinical trials