NCT05821959 Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
| NCT ID | NCT05821959 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Akouos, Inc. |
| Condition | Sensorineural Hearing Loss, Bilateral |
| Study Type | INTERVENTIONAL |
| Enrollment | 22 participants |
| Start Date | 2023-09-15 |
| Primary Completion | 2028-10 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 22 participants in total. It began in 2023-09-15 with a primary completion date of 2028-10.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
Part A of this trial will evaluate the safety and tolerability of a single surgical administration procedure in one or both ear(s) with one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos Delivery Device, together with the Precision Delivery Mechanism, to safely achieve the intended product performance.
Eligibility 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
Contact & Investigator
Peter Marks, MD
STUDY DIRECTOR
Eli Lilly and Company
Frequently Asked Questions
Who can join the NCT05821959 clinical trial?
This trial is open to participants of all sexes, studying Sensorineural Hearing Loss, Bilateral. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05821959 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05821959 currently recruiting?
Yes, NCT05821959 is actively recruiting participants. Contact the research team at AkouosClinicalTrials@Lilly.com for enrollment information.
Where is the NCT05821959 trial being conducted?
This trial is being conducted at San Francisco, United States, Iowa City, United States, Boston, United States, Cincinnati, United States and 5 additional locations.
Who is sponsoring the NCT05821959 clinical trial?
NCT05821959 is sponsored by Akouos, Inc.. The principal investigator is Peter Marks, MD at Eli Lilly and Company. The trial plans to enroll 22 participants.