Nemours Childrens Hospital

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations	Deafness, Autosomal Recessive 9, Ear Diseases, Hearing Loss, Sensorineural, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, auditory neuropathy, hearing disorder, hearing loss disorder, nervous system disorder, otorhinolaryngologic disease	Recruiting	Phase 1 / Phase 2	2026-08-05		NCT05788536
Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta	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 imperfecta	Active, not recruiting	Phase 3	2026-05-27		NCT05972551

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