Gene therapy for vestibular schwannoma treatment
A Phase 1/2 Trial of AAVAnc80-antiVEGF Gene Therapy in Individuals With Unilateral Vestibular Schwannoma
This study is testing a new gene therapy to see if it can safely help people with growing vestibular schwannomas and severe hearing loss.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 27 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Akouos, Inc. Industry-sponsored |
| Drugs / interventions | radiation |
| Locations | 4 sites (Baltimore, Maryland and 3 other locations) |
| Trial ID | NCT06517888 on ClinicalTrials.gov |
What this trial studies
This trial evaluates the safety and tolerability of a single unilateral administration of AAVAnc80-antiVEGF at three different dose levels. It also assesses the Akouos delivery device's effectiveness in achieving the desired therapeutic outcome. The focus is on patients with unilateral, progressive vestibular schwannoma larger than 2 mm and associated profound hearing loss. Participants will be monitored for their compliance with trial requirements and will be invited to join a long-term follow-up study.
Who should consider this trial
Good fit: Ideal candidates are individuals with unilateral, progressive vestibular schwannoma larger than 2 mm and profound hearing loss in the affected ear.
Not a fit: Patients with bilateral vestibular schwannoma or those who have undergone prior surgery or radiation therapy for vestibular schwannoma may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could provide a novel treatment option for patients suffering from vestibular schwannoma and associated hearing loss.
How similar studies have performed: Other studies have explored gene therapy approaches for similar conditions, but this specific application of anti-VEGF gene therapy is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Baltimore, Maryland and 3 other locations
- Johns Hopkins Hospital — Baltimore, Maryland, United States (Recruiting)
- Mayo Clinic — Rochester, Minnesota, United States (Recruiting)
- Vanderbilt Bill Wilkerson Center — Nashville, Tennessee, United States (Recruiting)
- University of Texas Southwestern — Dallas, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Akouos Clinical Trials
- Email: AkouosClinicalTrials@Lilly.com
- Phone: +1 (857) 410-1816
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.