Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorAkouos, Inc.
About this trial
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.
Eligibility criteria
Qualifiers
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)
Disqualifiers
None
Trial design
Treatments tested in this trial
- AAVAnc80-hOTOF via Akouos Delivery Device
- AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism
Treatment groups
22 Participants
are divided into 3 treatment groupsLocations
9Canada
The Hospital for Sick Children [email protected] +1 (416) 813 6683M5G 1X8, TorontoOntario, Canada
Taiwan
National Taiwan University Hospital100, Taipei Taiwan
United Kingdom
University College LondonNW1 2PG, LondonUnited Kingdom, United Kingdom
United States
California
University of California, San Francisco Benioff Children's Hospital94158, San Francisco United States
Sponsors and collaborators
Akouos, Inc.
Lead sponsor
Eli Lilly and Company
Collaborator
Source ClinicalTrials.gov