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NCT06517888PHASE1, PHASE2Recruiting

Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma

Akouos, Inc.

Start Date

1/17/2025

Completion Date

8/1/2029

Summary

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.

Eligibility Criteria

Age Range: 18 years to No maximum

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.

Interventions

COMBINATION_PRODUCT

AAVAnc80-antiVEGF via Akouos Delivery Device

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Conditions

Vestibular Schwannoma

Locations

Johns Hopkins Hospital

Baltimore, Maryland 21205

United States

Mayo Clinic

Rochester, Minnesota 55905

United States

Vanderbilt Bill Wilkerson Center

Nashville, Tennessee 37232

United States

University of Texas Southwestern

Dallas, Texas 75390

United States