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NCT05821959 · ClinicalTrials.gov registry record · Phase 1

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

A Phase 1 study of Sensorineural Hearing Loss, Bilateral, sponsored by Akouos.

Recruiting
Registry status
Phase 1
Development phase
22
Enrollment target
9
Study locations

NCT05821959: Recruiting Phase 1 study of Sensorineural Hearing Loss, Bilateral, sponsored by Akouos.

NCT05821959 is a Phase 1 study of Sensorineural Hearing Loss, Bilateral that is actively recruiting participants, run by Akouos. The registered enrollment target is 22 participants, below the 102-participant average among 4 other Sensorineural Hearing Loss, Bilateral trials with a reported enrollment target (78% lower). The trial reports 9 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT05821959, a Phase 1 study of Sensorineural Hearing Loss, Bilateral, is actively recruiting participants, sponsored by Akouos.

RECRUITING
Registry status
Phase 1
Development phase
22 participants
Enrollment target
9
Study locations

Study Summary

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.

Primary Outcome

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

Interventions

  • COMBINATION_PRODUCT AAVAnc80-hOTOF via Akouos Delivery Device
  • COMBINATION_PRODUCT AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism

Study Locations (9)

California

  • University of California, San Francisco Benioff Children's Hospital - San Francisco

Iowa

  • University of Iowa - Iowa City

Massachusetts

  • Boston Children's Hospital - Boston

Ohio

  • Cincinnati Children's Hospital Medical Center - Cincinnati

Pennsylvania

  • Children's Hospital of Philadelphia - Philadelphia

Tennessee

  • Vanderbilt Bill Wilkerson Center - Nashville

Ontario

  • The Hospital for Sick Children Jaina.negandhi@sickkids.ca +1 (416) 813 6683 - Toronto

Other

  • National Taiwan University Hospital - Taipei

Trial Details

FieldValue
Enrollment Target 22 participants
Start Date 2023-09-15
Est. Completion 2028-10
Phase Phase 1
Akouos

3 total trials

What NCT05821959 shows while recruiting

NCT05821959 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 22 participants, a relatively small participant target, below the 102-participant average among 4 other Sensorineural Hearing Loss, Bilateral trials with a reported enrollment target (78% lower).

The record links to 1 condition, with Sensorineural Hearing Loss, Bilateral appearing as the primary indexed condition, and to 2 interventions - of which AAVAnc80-hOTOF via Akouos Delivery Device is the first listed.

NCT05821959 lists 9 locations in 9 states (California, Iowa, Massachusetts).

Frequently Asked Questions

What is clinical trial NCT05821959 about?

NCT05821959 is a clinical study titled "Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss". 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 intende...

What is the current status of trial NCT05821959?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 22 participants. The study started on 2023-09-15. Estimated completion is 2028-10.

What conditions does trial NCT05821959 study?

This clinical trial studies the following conditions: Sensorineural Hearing Loss, Bilateral.

What interventions are being tested in trial NCT05821959?

The interventions under investigation include: AAVAnc80-hOTOF via Akouos Delivery Device (COMBINATION_PRODUCT), AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism (COMBINATION_PRODUCT).

Who is sponsoring clinical trial NCT05821959?

This trial is sponsored by Akouos, which has 3 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT05821959 being conducted?

This trial has 9 study locations across California, Iowa, Massachusetts, Ohio, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Source: ClinicalTrials.gov NCT05821959, the US trial registry maintained by the National Library of Medicine. NCT05821959 (small enrollment · multi site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.