Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Recruiting
Phase 1
Phase 2
Interventional Study
Sensorineural Hearing Loss, Bilateral
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 22 (estimated)
- Sponsor
- Akouos, Inc. · Industry
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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.
Trial Locations
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Eligibility Criteria
Criteria for Inclusion:
1. Participants may be of any age, based on Cohort Criteria
2. At least two 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…
Contacts