RecruitingPhase 1Phase 2NCT06517888

Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma

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


Sponsor

Akouos, Inc.

Enrollment

27 participants

Start Date

Jan 17, 2025

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 18 Years

Inclusion Criteria4

  • Presence of unilateral, progressive vestibular schwannoma.
  • Vestibular schwannoma larger than 2 mm.
  • Profound hearing loss, defined by pure-tone audiometry thresholds or word recognition score, in the affected ear.
  • 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.

Exclusion Criteria5

  • Prior diagnosis of NF2 and/or bilateral vestibular schwannoma.
  • Prior surgery or radiation therapy for vestibular schwannoma.
  • Clinical history consistent with endolymphatic hydrops (documented fluctuating sensorineural hearing loss and/or episodic vertigo) in the affected ear.
  • Profound hearing loss, defined by pure-tone audiometry thresholds or word recognition score, in the unaffected ear.
  • 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.

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Interventions

COMBINATION_PRODUCTAAVAnc80-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


Locations(4)

Johns Hopkins Hospital

Baltimore, Maryland, United States

Mayo Clinic

Rochester, Minnesota, United States

Vanderbilt Bill Wilkerson Center

Nashville, Tennessee, United States

University of Texas Southwestern

Dallas, Texas, United States

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NCT06517888


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