Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma

NCT ID: NCT06517888

Last Updated: 2025-10-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

27 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-01-17

Study Completion Date

2029-08-31

Brief Summary

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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.

Detailed Description

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Conditions

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Vestibular Schwannoma

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

AAVAnc80-antiVEGF via Akouos Delivery Device

Intervention Type COMBINATION_PRODUCT

AAVAnc80-antiVEGF is a sterile suspension intended to be administered via intracochlear administration as a single dose using the Akouos Delivery Device

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.

Group Type EXPERIMENTAL

AAVAnc80-antiVEGF via Akouos Delivery Device

Intervention Type COMBINATION_PRODUCT

AAVAnc80-antiVEGF is a sterile suspension intended to be administered via intracochlear administration as a single dose using the Akouos Delivery Device

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

Group Type EXPERIMENTAL

AAVAnc80-antiVEGF via Akouos Delivery Device

Intervention Type COMBINATION_PRODUCT

AAVAnc80-antiVEGF is a sterile suspension intended to be administered via intracochlear administration as a single dose using the Akouos Delivery Device

Interventions

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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

Intervention Type COMBINATION_PRODUCT

Eligibility Criteria

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Inclusion Criteria

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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Eli Lilly and Company

INDUSTRY

Sponsor Role collaborator

Akouos, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Kathy Reape, MD

Role: STUDY_DIRECTOR

Akouos, Inc.

Locations

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Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status RECRUITING

Mayo Clinic

Rochester, Minnesota, United States

Site Status RECRUITING

Vanderbilt Bill Wilkerson Center

Nashville, Tennessee, United States

Site Status RECRUITING

University of Texas Southwestern

Dallas, Texas, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Akouos Clinical Trials

Role: CONTACT

+1 (857) 410-1816

Facility Contacts

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Role: primary

+1 (410) 955 3157

Dan Osland

Role: primary

+1 507-266-1470

Taha Jan, MD

Role: primary

+1 (615) 322-4386

Role: primary

+1 (214) 648-3626

Other Identifiers

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AK-antiVEGF-101

Identifier Type: -

Identifier Source: org_study_id

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