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

ArmDescriptionAssigned 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
  • Combination Product: AAVAnc80-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.
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
  • Combination Product: AAVAnc80-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.

Recruiting Locations

Vanderbilt Bill Wilkerson Center
Nashville, Tennessee 37232
Contact:
Taha Jan, MD
615-421-1228
taha.a.jan@vumc.org

More Details

Status
Recruiting
Sponsor
Akouos, Inc.

Study Contact

Akouos Clinical Trials
+1 (857) 410-1816
AkouosClinicalTrials@Lilly.com

Detailed Description

The following two dose levels will be tested sequentially: - 4.1E11 total vg/cochlea - 8.1E11 total vg/cochlea