Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma

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.

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.