Vestibular SchwannomaPhase 1/2NCT06517888
Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma
A Phase 1/2 study testing AAVAnc80-antiVEGF via Akouos Delivery Device in people with Vestibular Schwannoma, run by Eli Lilly and Company at 4 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Vestibular Schwannoma
- What is being tested?
- AAVAnc80-antiVEGF via Akouos Delivery Device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 4 sitesUnited States
- Study dates
- 17 Jan 2025to Aug 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Johns Hopkins Hospital
Baltimore, Maryland, 21205
410-955-3157, ototrials@live.johnshopkins.edu
- Recruiting
University of Texas Southwestern
Dallas, Texas, 75390
214-648-3626, OTOResearch@UTSouthwestern.edu
- Recruiting
Vanderbilt Bill Wilkerson Center
Nashville, Tennessee, 37232
Taha Jan, MD, 615-322-4386, Taha.a.jan@vumc.org
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Dan Osland, 507-266-1470, Osland.daniel@mayo.edu
About this study
Official title: 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.
- Conditions
- Vestibular Schwannoma
- Being tested
- AAVAnc80-antiVEGF via Akouos Delivery Device
- Keywords
- Vestibular schwannoma, Hearing loss, Acoustic neuroma, anti-VEGF
- Medical subject headings
- Neuroma, Acoustic, Hearing Loss
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAVAnc80-antiVEGF via Akouos Delivery DeviceCombination product
AAVAnc80-antiVEGF is a sterile suspension intended to be administered via intracochlear administration as a single dose using the Akouos Delivery Device
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Adult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 1) in the study ear using a sterile, one-time use investigational medical device. Receives: Combination Product: AAVAnc80-antiVEGF via Akouos Delivery Device |
| Cohort 2 | Experimental | Adult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 2) in the study ear using a sterile, one-time use investigational medical device. Receives: Combination Product: AAVAnc80-antiVEGF via Akouos Delivery Device |
| Cohort 3 | Experimental | Adult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 3) in the study ear using a sterile, one-time use investigational medical device Receives: Combination Product: AAVAnc80-antiVEGF via Akouos Delivery Device |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Adverse Events (AEs)
AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery device)
Measured: through trial completion, approximately one year
Secondary outcomes (2)
Tumor Volume
Change from baseline in tumor size via MRI (volumetric analysis)
Measured: through trial completion, approximately one year
Performance of Akouos Delivery Device
Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-antiVEGF to the intracochlear space
Measured: through trial completion, approximately one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2024
- Study start17 Jan 2025
- Primary completionAug 2029 (estimated)
- Study completionAug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Vestibular SchwannomaStudies of the same condition, open and under way.
- More trials run by Eli Lilly and CompanyThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eli Lilly and Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Aaron Tward, MD, Study director, Eli Lilly and Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06517888
- Sponsor's protocol ID
- AK-antiVEGF-101
- Status last verified
- Nov 2026
- First submitted
- 19 Jul 2024
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Trial of AAVAnc80-antiVEGF Gene Therapy in Individuals With Unilateral Vestibular Schwannoma
- ClinicalTrials.gov ID
- NCT06517888
- Acronym / sponsor ID
- AK-antiVEGF-101
- Conditions
- Vestibular Schwannoma
- Keywords
- Vestibular schwannoma, Hearing loss, Acoustic neuroma, anti-VEGF
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 27 participants (estimated)
- Lead sponsor
- Eli Lilly and CompanyIndustry (pharma, biotech, devices)
- Study start
- 17 Jan 2025 (actual)
- Primary completion
- Aug 2029 (estimated)
- Study completion
- Aug 2029 (estimated)
- First posted
- 24 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Combination Product: AAVAnc80-antiVEGF via Akouos Delivery DeviceAdult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 1) in the study ear using a sterile, one-time use investigational medical device. |
| Cohort 2 | Experimental | Combination Product: AAVAnc80-antiVEGF via Akouos Delivery DeviceAdult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 2) in the study ear using a sterile, one-time use investigational medical device. |
| Cohort 3 | Experimental | Combination Product: AAVAnc80-antiVEGF via Akouos Delivery DeviceAdult participants to receive a single unilateral intracochlear administration of AAVAnc80-antiVEGF (dose level 3) in the study ear using a sterile, one-time use investigational medical device |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAVAnc80-antiVEGF via Akouos Delivery DeviceAAVAnc80-antiVEGF is a sterile suspension intended to be administered via intracochlear administration as a single dose using the Akouos Delivery Device | Combination product | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
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)
2 secondary outcomes
Tumor Volume
Time frame: through trial completion, approximately one year
Performance of Akouos Delivery Device
Time frame: through trial completion, approximately one year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2026.
Related trials for Vestibular Schwannoma
Enrolling by invitationObservational study
Long Term Follow-up Study of AAVAnc80-antiVEGF Gene Therapy
Vestibular Schwannoma
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Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)
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Steroids for Facial Nerve Function Protection in Post-surgical Vestibular Schwannoma Patients
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Vestibular Schwannoma, Unilateral Vestibular Deficit
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Aaron Tward, MD study director
Eli Lilly and Company
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AAVAnc80-antiVEGF via Akouos Delivery Device in people with Vestibular Schwannoma, run by Eli Lilly and Company at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lilly Clinical Trials (+1 (857) 410-1816, AkouosClinicalTrials@Lilly.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of Adverse Events (AEs) (time frame: through trial completion, approximately one year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2026.