Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Sensorineural Hearing Loss, Bilateral
About this trial
This is an interventional treatment trial for Sensorineural Hearing Loss, Bilateral focused on measuring Otoferlin, Auditory neuropathy (AN), Deafness, autosomal recessive 9 (DFNB9), Hearing loss, Hearing loss, bilateral, Hearing loss, sensorineural
Eligibility Criteria
Inclusion Criteria: Age 7 to 17 years old (inclusive) for Cohort #1a; age 2 to 17 years old (inclusive) at the time of AAVAnc80-hOTOF administration for Cohorts #1b and #2 Biallelic 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 Bilateral cochlear implants 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
Sites / Locations
- University of IowaRecruiting
- Children's Hospital of PhiladelphiaRecruiting
- National Taiwan University HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Cohort 1a and Cohort 1b
Cohort 2
Cohort 1a: 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: Participants aged 2 to 17 years old (inclusive) at the time of AAVAnc80-hOTOF administration to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device
Cohort 2: Participants aged 2 to 17 years old (inclusive) at the time of AAVAnc80-hOTOF administration to receive intracochlear administration of AAVAnc80-hOTOF (dose level 2) in the study ear using a sterile, one-time use investigational medical device