RecruitingPhase 1/2

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

United States22 participantsStarted 2023-09-15

Plain-language 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.

Who can participate

SexALL

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Inclusion criteria

✓. Participants may be of any age, based on Cohort Criteria
✓. At least two mutations in the otoferlin gene
✓. Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR
✓. Preserved distortion product otoacoustic emissions (DPOAEs)
✓. 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

✕. 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
✕. Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF
✕. 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

What they're measuring

Frequency of Adverse Events (AEs)

Timeframe: through trial completion, approximately 1 year

Trial details

NCT IDNCT05821959

SponsorAkouos, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-10

Contact for this trial

Akouos Clinical Trials

+1 857-410-1816 AkouosClinicalTrials@Lilly.com

View on ClinicalTrials.gov More Sensorineural Hearing Loss, Bilateral trials