Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma

April 1, 2026 updated by: Akouos, Inc.

A Phase 1/2 Trial of AAVAnc80-antiVEGF Gene Therapy in Individuals With Unilateral Vestibular Schwannoma

This trial will evaluate the safety and tolerability of a single unilateral administration of one of three dose levels of AAVAnc80-antiVEGF and will evaluate the Akouos delivery device to safely achieve the intended product performance.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

27

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Maryland
      • Baltimore, Maryland, United States, 21205
    • Minnesota
      • Rochester, Minnesota, United States, 55905
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt Bill Wilkerson Center
        • Contact:
    • Texas
      • Dallas, Texas, United States, 75390

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Criteria for Inclusion:

  1. Presence of unilateral, progressive vestibular schwannoma.
  2. Vestibular schwannoma larger than 2 mm.
  3. Profound hearing loss, defined by pure-tone audiometry thresholds or word recognition score, in the affected ear.
  4. Able and willing to comply with all trial requirements, including willingness to participate in a separate long term follow-up study after completion of this trial.

Criteria for Exclusion:

  1. Prior diagnosis of NF2 and/or bilateral vestibular schwannoma.
  2. Prior surgery or radiation therapy for vestibular schwannoma.
  3. Clinical history consistent with endolymphatic hydrops (documented fluctuating sensorineural hearing loss and/or episodic vertigo) in the affected ear.
  4. Profound hearing loss, defined by pure-tone audiometry thresholds or word recognition score, in the unaffected ear.
  5. Prior participation in a clinical trial with an investigational drug within six months prior to administration (Day 0), or any prior participation in a gene therapy clinical trial.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1
Adult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 1) in the study ear using a sterile, one-time use investigational medical device.
Combination product: AAVAnc80-antiVEGF via Akouos Delivery Device
AAVAnc80-antiVEGF is a sterile suspension intended to be administered via intracochlear administration as a single dose using the Akouos Delivery Device
Experimental: Cohort 2
Adult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 2) in the study ear using a sterile, one-time use investigational medical device.
Combination product: AAVAnc80-antiVEGF via Akouos Delivery Device
AAVAnc80-antiVEGF is a sterile suspension intended to be administered via intracochlear administration as a single dose using the Akouos Delivery Device
Experimental: Cohort 3
Adult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 3) in the study ear using a sterile, one-time use investigational medical device
Combination product: AAVAnc80-antiVEGF via Akouos Delivery Device
AAVAnc80-antiVEGF is a sterile suspension intended to be administered via intracochlear administration as a single dose using the Akouos Delivery Device

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frequency of Adverse Events (AEs)
Time Frame: through trial completion, approximately one year
AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery device)
through trial completion, approximately one year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Tumor Volume
Time Frame: through trial completion, approximately one year
Change from baseline in tumor size via MRI (volumetric analysis)
through trial completion, approximately one year
Performance of Akouos Delivery Device
Time Frame: through trial completion, approximately one year
Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-antiVEGF to the intracochlear space
through trial completion, approximately one year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Akouos, Inc.

Collaborators

Eli Lilly and Company

Investigators

  • Study Director: Aaron Tward, MD, Eli Lilly and Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 17, 2025

Primary Completion (Estimated)

August 1, 2029

Study Completion (Estimated)

August 1, 2029

Study Registration Dates

First Submitted

July 19, 2024

First Submitted That Met QC Criteria

July 19, 2024

First Posted (Actual)

July 24, 2024

Study Record Updates

Last Update Posted (Actual)

April 6, 2026

Last Update Submitted That Met QC Criteria

April 1, 2026

Last Verified

October 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AK-antiVEGF-101

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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