A Phase 1/2 Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Biallelic Otoferlin Gene Mutations

AK-OTOF-101

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.

2023-09-15

2028-10-01

2028-10-01

Recruiting

Early phase I

14

Inclusion Criteria: 1. Age 2 to 17 years old (inclusive) at the time of AAVAnc80-hOTOF administration 2. Biallelic 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 Exclusion Criteria: 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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 14, 'type': 'ESTIMATED'}}

2024-02-08