Health TrialsbyHealthPanel

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

ConditionSensorineural Hearing Loss, Bilateral
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

This trial does not accept healthy volunteers

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

Design model

Sequential

Treatments tested in this trial

  • 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.

Treatment groups

22 Participants
are divided into 3 treatment groups
Group A: Cohort 1a and Cohort 1bExperimental treatment 1 intervention
Group B: Cohort 2Experimental treatment 1 intervention
Group C: Bilateral ExpansionExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Frequency of Adverse Events (AEs)

AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery system)

Time frame
through trial completion, approximately 1 year

Secondary outcomes

1

Auditory Brainstem Response (ABR) Threshold

Changes from baseline in ABR intensity threshold (decibels normal hearing level \[dB nHL\])

Time frame
through trial completion, approximately 1 year
2

Performance of the Akouos Delivery Device and Precision Delivery Mechanism

Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-hOTOF to the intracochlear space

Time frame
through trial completion, approximately 1 year

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Akouos, Inc.

Lead sponsor

Eli Lilly and Company

Collaborator

Source ClinicalTrials.gov
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