Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

Sensorineural Hearing Loss, Bilateral Clinical Trial

Official title:

A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05821959
• Other study ID #: AK-OTOF-101
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: September 15, 2023
• Est. completion date: October 2028

Study information

• Verified date: May 2024
• Source: Akouos, Inc.
• Contact: Akouos Clinical Trials
• Phone: +1 (857) 410-1816
• Email: clinicaltrials[@]akouos.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Description:

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 14
• Est. completion date: October 2028
• Est. primary completion date: October 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Deafness
• Hearing Loss
• Hearing Loss, Bilateral
• Hearing Loss, Sensorineural
• Sensorineural Hearing Loss, Bilateral

Intervention

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.

Locations

Country	Name	City	State
Taiwan	National Taiwan University Hospital	Taipei City
United Kingdom	University College London	London
United States	University of Iowa	Iowa City	Iowa
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (2)

Lead Sponsor	Collaborator
Akouos, Inc.	Eli Lilly and Company

Countries where clinical trial is conducted

United States,  Taiwan,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Frequency of Adverse Events (AEs)	AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery device)	through trial completion, approximately 1 year
Secondary	Auditory Brainstem Response (ABR) Threshold	Changes from baseline in ABR intensity threshold (decibels normal hearing level [dB nHL])	through trial completion, approximately 1 year
Secondary	Performance of the Akouos Delivery Device	Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-hOTOF to the intracochlear space	through trial completion, approximately 1 year