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Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

Primary Purpose

Sensorineural Hearing Loss, Bilateral

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
AAVAnc80-hOTOF via Akouos Delivery Device
Sponsored by
Akouos, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

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

2 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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

Arm Type

Experimental

Experimental

Arm Label

Cohort 1a and Cohort 1b

Cohort 2

Arm Description

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

Outcomes

Primary Outcome Measures

Frequency of Adverse Events (AEs)
AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery device)

Secondary Outcome Measures

Auditory Brainstem Response (ABR) Threshold
Changes from baseline in ABR intensity threshold (decibels normal hearing level [dB nHL])
Performance of the Akouos Delivery Device
Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-hOTOF to the intracochlear space

Full Information

First Posted
February 8, 2023
Last Updated
October 10, 2023
Sponsor
Akouos, Inc.
Collaborators
Eli Lilly and Company
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1. Study Identification

Unique Protocol Identification Number
NCT05821959
Brief Title
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Official Title
A Phase 1/2 Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Biallelic Otoferlin Gene Mutations
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 15, 2023 (Actual)
Primary Completion Date
October 2028 (Anticipated)
Study Completion Date
October 2028 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Akouos, Inc.
Collaborators
Eli Lilly and Company

4. Oversight

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
Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sensorineural Hearing Loss, Bilateral
Keywords
Otoferlin, Auditory neuropathy (AN), Deafness, autosomal recessive 9 (DFNB9), Hearing loss, Hearing loss, bilateral, Hearing loss, sensorineural

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
14 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1a and Cohort 1b
Arm Type
Experimental
Arm Description
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
Arm Title
Cohort 2
Arm Type
Experimental
Arm Description
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
Intervention Type
Combination Product
Intervention Name(s)
AAVAnc80-hOTOF via Akouos Delivery Device
Intervention Description
AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.
Primary Outcome Measure Information:
Title
Frequency of Adverse Events (AEs)
Description
AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery device)
Time Frame
through trial completion, approximately 2 years
Secondary Outcome Measure Information:
Title
Auditory Brainstem Response (ABR) Threshold
Description
Changes from baseline in ABR intensity threshold (decibels normal hearing level [dB nHL])
Time Frame
through trial completion, approximately 2 years
Title
Performance of the Akouos Delivery Device
Description
Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-hOTOF to the intracochlear space
Time Frame
through trial completion, approximately 2 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Akouos Clinical Trials
Phone
+1 (857) 410-1816
Email
clinicaltrials@akouos.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kathy Reape, MD
Organizational Affiliation
Akouos, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kesten Anderson
Phone
319-356-8320
Email
Kesten-Anderson@uiowa.edu
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19146
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Aminat Adegbuji
Phone
267-426-8380
Email
adegbujia@chop.edu
Facility Name
National Taiwan University Hospital
City
Taipei City
ZIP/Postal Code
100
Country
Taiwan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Wan-Cian Liao
Phone
+886- 953-728-657
Email
mulong0328@gmail.com

12. IPD Sharing Statement

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Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

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