Addenbrooke's Hospital, Cambridge University Hospitals NHS FT

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B	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 disorder	Recruiting	Phase 1 / Phase 2	2026-10-08		NCT06379789
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

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