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Recruiting NCT05821959

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

Phase I / Phase II Interventional Sensorineural Hearing Loss, Bilateral

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: AAVAnc80-hOTOF via Akouos Delivery Device, AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism.
Who it may be relevant to
Registry conditions: Sensorineural Hearing Loss, Bilateral. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Canada, Taiwan, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Detailed description

The following two dose levels will be tested:

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

Interventions

  • 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.
  • Combination product AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism
    AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time bilateral intracochlear administration using the Akouos Delivery Device together with the Precision Delivery Mechanism.

Primary outcome measures

  • Frequency of Adverse Events (AEs) [Time frame: through trial completion, approximately 1 year]
Secondary outcome measures (2)
  • Auditory Brainstem Response (ABR) Threshold [Time frame: through trial completion, approximately 1 year]
  • Performance of the Akouos Delivery Device and Precision Delivery Mechanism [Time frame: through trial completion, approximately 1 year]

Eligibility criteria

Criteria for Inclusion:

  • 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

Criteria for Exclusion:

  • 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 6 centers
  • University of California, San Francisco Benioff Children's Hospital — San Francisco
  • University of Iowa — Iowa City
  • Boston Children's Hospital — Boston
  • Cincinnati Children's Hospital Medical Center — Cincinnati
  • Children's Hospital of Philadelphia — Philadelphia
  • Vanderbilt Bill Wilkerson Center — Nashville
Canada · 1 center
  • The Hospital for Sick Children Jaina.negandhi@sickkids.ca +1 (416) 813 6683 — Toronto
Taiwan · 1 center
  • National Taiwan University Hospital — Taipei
United Kingdom · 1 center
  • University College London — London

Identifiers

NCT: NCT05821959 · AK-OTOF-101

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗