Clinical trial NCT05821959: Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Official title: A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Sensorineural Hearing Loss, Bilateral
Keywords: Otoferlin
Auditory neuropathy (AN)
Deafness, autosomal recessive 9 (DFNB9)
Hearing loss
Hearing loss, bilateral
Hearing loss, sensorineural
Lead sponsor: Akouos, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Eli Lilly and Company
First posted: 2023-04-20
Enrollment: 14
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief 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.

Detailed description: The following two dose levels will be tested sequentially:

* 4.1E11 total vg/cochlea
* 8.1E11 total vg/cochlea

Eligibility 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

Primary outcomes:
measure: Frequency of Adverse Events (AEs)
timeFrame: through trial completion, approximately 1 year
Secondary outcomes:
measure: Auditory Brainstem Response (ABR) Threshold
timeFrame: through trial completion, approximately 1 year
measure: Performance of the Akouos Delivery Device
timeFrame: through trial completion, approximately 1 year
Other outcomes:
Locations:
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt Bill Wilkerson Center
state: Tennessee
city: Toronto
country: Canada
facility: The Hospital for Sick Children Jaina.negandhi@sickkids.ca +1 (416) 813 6683
state: Ontario
city: Taipei City
country: Taiwan
facility: National Taiwan University Hospital
city: London
country: United Kingdom
facility: University College London
Study officials:
affiliation: Akouos, Inc.
name: Kathy Reape, MD
role: STUDY_DIRECTOR
References: