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Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05821959
Recruitment Status : Recruiting
First Posted : April 20, 2023
Last Update Posted : February 8, 2024
Sponsor:
Collaborator:
Eli Lilly and Company
Information provided by (Responsible Party):
Akouos, Inc.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Sensorineural Hearing Loss, Bilateral Combination Product: AAVAnc80-hOTOF via Akouos Delivery Device Phase 1 Phase 2

Detailed Description:

The following two dose levels will be tested sequentially:

  • 4.1E11 total vg/cochlea
  • 8.1E11 total vg/cochlea
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 14 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Biallelic Otoferlin Gene Mutations
Actual Study Start Date : September 15, 2023
Estimated Primary Completion Date : October 2028
Estimated Study Completion Date : October 2028

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Cohort 1a and Cohort 1b

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

 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.
Experimental: Cohort 2
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
 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.


Outcome Measures
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Primary Outcome Measures :
  1. Frequency of Adverse Events (AEs) [ Time Frame: through trial completion, approximately 2 years ]
    AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery device)


Secondary Outcome Measures :
  1. Auditory Brainstem Response (ABR) Threshold [ Time Frame: through trial completion, approximately 2 years ]
    Changes from baseline in ABR intensity threshold (decibels normal hearing level [dB nHL])

  2. Performance of the Akouos Delivery Device [ Time Frame: through trial completion, approximately 2 years ]
    Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-hOTOF to the intracochlear space


Eligibility Criteria
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Ages Eligible for Study:   2 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age 2 to 17 years old (inclusive) at the time of AAVAnc80-hOTOF administration
  2. Biallelic 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

Exclusion Criteria:

  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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05821959


Contacts
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Contact: Akouos Clinical Trials +1 (857) 410-1816 clinicaltrials@akouos.com

Locations
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United States, Iowa
University of Iowa Recruiting
Iowa City, Iowa, United States, 52242
Contact: Kesten Anderson    319-356-8320    Kesten-Anderson@uiowa.edu   
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19146
Contact: Aminat Adegbuji    267-426-8380    adegbujia@chop.edu   
Taiwan
National Taiwan University Hospital Recruiting
Taipei City, Taiwan, 100
Contact: Wan-Cian Liao    +886- 953-728-657    mulong0328@gmail.com   
Sponsors and Collaborators
Akouos, Inc.
Eli Lilly and Company
Investigators
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Study Director: Kathy Reape, MD Akouos, Inc.
More Information
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Responsible Party: Akouos, Inc.
ClinicalTrials.gov Identifier: NCT05821959    
Other Study ID Numbers: AK-OTOF-101
First Posted: April 20, 2023    Key Record Dates
Last Update Posted: February 8, 2024
Last Verified: February 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: Yes
Keywords provided by Akouos, Inc.:
Otoferlin
Auditory neuropathy (AN)
Deafness, autosomal recessive 9 (DFNB9)
 Hearing loss
Hearing loss, bilateral
Hearing loss, sensorineural
Additional relevant MeSH terms:
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Hearing Loss
Deafness
Hearing Loss, Sensorineural
Hearing Loss, Bilateral
Hearing Disorders
 Ear Diseases
Otorhinolaryngologic Diseases
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases