Gene therapy for hearing loss caused by otoferlin gene mutations
A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations
This study is testing a new gene therapy to see if it can help restore hearing in people with severe hearing loss caused by mutations in the otoferlin gene.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 22 (estimated) |
| Sex | All |
| Sponsor | Akouos, Inc. Industry-sponsored |
| Locations | 9 sites (San Francisco, California and 8 other locations) |
| Trial ID | NCT05821959 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the safety and tolerability of a gene therapy treatment for profound bilateral sensorineural hearing loss caused by mutations in the otoferlin gene. Participants will receive a single unilateral administration of one of two dose levels of the AAVAnc80-hOTOF gene therapy via the Akouos delivery device. The study will assess the effectiveness of this approach in restoring hearing function in affected individuals. The trial is divided into two phases, focusing on different dose levels to determine the optimal treatment strategy.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals of any age with at least two mutations in the otoferlin gene and profound bilateral sensorineural hearing loss.
Not a fit: Patients with persistent ear infections, anatomical abnormalities of the ear, or those with bilateral cochlear implants may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could significantly improve hearing in patients with otoferlin gene-mediated hearing loss.
How similar studies have performed: Other studies have shown promise in gene therapy approaches for hearing loss, but this specific application of otoferlin gene therapy is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Criteria for Inclusion: 1. Participants may be of any age, based on Cohort Criteria 2. At least two mutations in the otoferlin gene 3. Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR 4. Preserved distortion product otoacoustic emissions (DPOAEs) 5. Able and willing to comply with all study requirements, including willingness to participate in a separate long term follow-up study after completion of this trial Criteria for Exclusion: 1. Persistent ear infections, anatomic or other abnormalities of the ear, and/or medical conditions that would contraindicate undergoing surgery, anesthesia, and/or administration of investigational gene therapy 2. Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF 3. Prior participation in a clinical trial with an investigational drug, within six months prior to administration, or any prior participation in a gene therapy clinical trial
Where this trial is running
San Francisco, California and 8 other locations
- University of California, San Francisco Benioff Children's Hospital — San Francisco, California, United States (Recruiting)
- University of Iowa — Iowa City, Iowa, United States (Recruiting)
- Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
- Vanderbilt Bill Wilkerson Center — Nashville, Tennessee, United States (Recruiting)
- The Hospital for Sick Children Jaina.negandhi@sickkids.ca +1 (416) 813 6683 — Toronto, Ontario, Canada (Recruiting)
- National Taiwan University Hospital — Taipei, Taiwan (Recruiting)
- University College London — London, United Kingdom, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Akouos Clinical Trials
- Email: AkouosClinicalTrials@Lilly.com
- Phone: +1 857-410-1816
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.