NCT05788536 - A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

June 27, 2023

Primary Completion Date

April 19, 2031

Study Completion Date

April 19, 2031

Conditions

Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

Interventions

GENETIC

DB-OTO

"DB-OTO will be administered as a single intracochlear injection into one ear (Part A).~* LD Cohort (lower dose)~* HD Cohort (high dose) - not implemented"

GENETIC

DB-OTO

DB-OTO will be administered as a single intracochlear injection into both ears (Part B). For bilateral injections (Part B), patients will receive DB-OTO in 1 surgical session.

Trial Locations (15)

10032

RECRUITING

Columbia University Irving Medical Center, New York

28034

RECRUITING

Ramon y Cajal University Hospital, Madrid

31008

RECRUITING

Clinica Universidad de Navarra- Pamplona, Pamplona

32207

RECRUITING

Nemours Children s Clinic, Jacksonville

32827

RECRUITING

Nemours Childrens Hospital, Orlando

35016

WITHDRAWN

Hospital Universitario Materno Infantil en las Palmas de Gran Canaria, Las Palmas de Gran Canaria

45229

RECRUITING

Cincinnati Children's Hospital Medical Center, Cincinnati

53226

RECRUITING

Medical College of Wisconsin, Milwaukee

72076

RECRUITING

University Hospital Tubingen, Tübingen

90024

RECRUITING

University of California Los Angeles Medical Center, Los Angeles

92123

RECRUITING

Rady Children's Hospital, San Diego

98105

RECRUITING

Seattle Children's Hospital, Seattle

02115

RECRUITING

Boston Children's Hospital - Main, Boston

CB2 0QQ

RECRUITING

Addenbrooke's Hospital, Cambridge University Hospitals NHS FT, Cambridge

WC1N 3JH

RECRUITING

Great Ormond Street Hospital For Children NHS Foundation Trust, London

All Listed Sponsors

lead

Regeneron Pharmaceuticals

INDUSTRY