Sensorineural Hearing Loss, Bilateral
Conditions
Keywords
Otoferlin, Auditory neuropathy (AN), Deafness, autosomal recessive 9 (DFNB9), Hearing loss, Hearing loss, bilateral, Hearing loss, sensorineural
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.
Detailed description
The following two dose levels will be tested: * Up to 4.1E11 total vg/cochlea * Up to 8.1E11 total vg/cochlea
Interventions
COMBINATION_PRODUCTAAVAnc80-hOTOF via Akouos Delivery Device
AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.
COMBINATION_PRODUCTAAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism
AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time bilateral intracochlear administration using the Akouos Delivery Device together with the Precision Delivery Mechanism.
Sponsors
Akouos, Inc.
Eli Lilly and Company
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Frequency of Adverse Events (AEs) | through trial completion, approximately 1 year | AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery system) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Auditory Brainstem Response (ABR) Threshold | through trial completion, approximately 1 year | Changes from baseline in ABR intensity threshold (decibels normal hearing level \[dB nHL\]) |
| Performance of the Akouos Delivery Device and Precision Delivery Mechanism | through trial completion, approximately 1 year | Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-hOTOF to the intracochlear space |
Countries
Canada, Taiwan, United Kingdom, United States
Contacts
CONTACTAkouos Clinical Trials
STUDY_DIRECTORPeter Marks, MD
Eli Lilly and Company
Outcome results
None listed