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NCT Number: NCT06517888

Anti-VEGF Gene Therapy Trial for 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.

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Key information

Conditions

Vestibular Schwannoma Cranial Nerve Diseases Cranial Nerve Neoplasms Ear Diseases Hearing Disorders Hearing Loss Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Germ Cell and Embryonal Neoplasms, Nerve Tissue Nerve Sheath Neoplasms Nervous System Diseases Nervous System Neoplasms Neurilemmoma Neuroectodermal Tumors Neuroendocrine Tumors Neurologic Manifestations Neuroma Neuroma, Acoustic Otorhinolaryngologic Diseases Otorhinolaryngologic Neoplasms Pathological Conditions, Signs and Symptoms Peripheral Nervous System Neoplasms Retrocochlear Diseases Sensation Disorders Signs and Symptoms Vestibulocochlear Nerve Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Johns Hopkins Hospital, Baltimore, Maryland, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Criteria for Inclusion:

  • 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.

Criteria for Exclusion:

  • 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.

Treatment and study plan

AAVAnc80-antiVEGF via Akouos Delivery Device

Combination Product

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

Primary outcomes

  1. 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)

Secondary outcomes

  1. Tumor Volume

    Time frame: through trial completion, approximately one year

    Change from baseline in tumor size via MRI (volumetric analysis)

  2. 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

Study contacts

Contact information is provided by the study sponsor or research team.

Akouos Clinical Trials

CONTACT

[email protected]

+1 (857) 410-1816

Sponsors and collaborators

Lead sponsor

Akouos, Inc.

Industry

Collaborators

  • Eli Lilly and Company

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2029
Study completion
2029
First posted
Jul 24, 2024
Registry last updated
Apr 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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