Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma

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Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years and Over
Gender	All

More 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.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT06517888
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 1/Phase 2
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Akouos, Inc.
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Kathy Reape, MD
Principal Investigator Affiliation	Akouos, Inc.
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Recruiting
Countries	United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Vestibular Schwannoma

Arms & Interventions

Arms

Experimental: Cohort 1

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.

Experimental: Cohort 2

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.

Experimental: Cohort 3

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

Interventions

Combination Product: - AAVAnc80-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

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Mayo Clinic, Rochester, Minnesota

Status

Recruiting

Address

Mayo Clinic

Rochester, Minnesota, 55905

Site Contact

Amy Tuchscherer

[email protected]

+1 (507) 538-5862

Vanderbilt Bill Wilkerson Center, Nashville, Tennessee

Status