Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Purpose
Part A of this trial will evaluate the safety and tolerability of a single unilateral administration of one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos delivery device to safely achieve the intended product performance.
Condition
- Sensorineural Hearing Loss, Bilateral
Eligibility
- Eligible Ages
- All ages
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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. Bilateral cochlear implants
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
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Cohort 1a and Cohort 1b |
Cohort 1a: Two participants aged 7 to 17 years old (inclusive) to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device Cohort 1b: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age |
|
|
Experimental Cohort 2 |
Cohort 2: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 2) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age |
|
Recruiting Locations
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Akouos, Inc.
Detailed Description
The following two dose levels will be tested sequentially: - 4.1E11 total vg/cochlea - 8.1E11 total vg/cochlea