An Investigator Initiated Study for OTOV101N+OTOV101C Injection

Summary

This study is an investigator initiated study to evaluate the safety, tolerability, and efficacy of OTOV101N+OTOV101C injection in treating patients with OTOF mutation-related deafness. The enrolled subjects who meet the inclusion and exclusion criteria will receive the gene therapy of OTOV101N+OTOV101C injection via intracochlear injection. All participants will return to the hospital for safety and efficacy evaluations at predetermined time points defined by protocol during the study (Week 1 ± 1 Day, Week 2 ± 3 Days, Week 3 ± 3 Days, Month 1 ± 3 Days, Month 2 ± 3 Days, Month 4 ± 6 Days, Month 6 ± 6 Days, Month 9 ± 6 Days, Month 12 ± 6 Days/EOS (end of study)/unscheduled visit).

Timeline

Start

2023-06-26

Primary completion

2024-12-06

Completion

2025-02-18

Publications

• Qi J, Zhang L, Lu L, Tan F, Cheng C, Lu Y, Dong W, Zhou Y, Fu X, Jiang L, Tan C, Zhang S, Sun S, Song H, Duan M, Zha D, Sun Y, Gao X, Xu L, Zeng FG, Chai R. AAV gene therapy for autosomal recessive deafness 9: a single-arm trial. Nat Med. 2025 Sep;31(9):2917-2926. doi: 10.1038/s41591-025-03773-w. Epub 2025 Jul 2.

Drugs

Indications