Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

Conditions

• Sensorineural Hearing Loss, Bilateral

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Interventions

• AAVAnc80-hOTOF via Akouos Delivery Device — COMBINATION_PRODUCT
  AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.
• AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism — COMBINATION_PRODUCT
  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.

Study Details

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.

Key Dates

Start date

Sep 15, 2023

Status verified

Dec 2025

Primary completion

Oct 31, 2028

Completion

Oct 31, 2028

Study Design

Enrollment

22 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1a and Cohort 1b
  Cohort 1a: Two 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: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age
• Experimental: Cohort 2
  Cohort 2: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 2) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age
• Experimental: Bilateral Expansion
  Bilateral Expansion: up to eight eligible participants of any age, and no cochlear implants, may be eligible for enrollment into the Bilateral Expansion Cohort (simultaneous bilateral administration of AAVAnc80-hOTOF).

Primary Outcome Measure

Frequency of Adverse Events (AEs) [ Time Frame: through trial completion, approximately 1 year ]

Central Contacts

• Akouos Clinical Trials
  +1 857-410-1816
  [email protected]

Locations (6)

Find similar trials in San Francisco, CA

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