Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma

Phase 1

Recruiting

• Conditions: Vestibular Schwannoma
• Interventions: Combination Product: AAVAnc80-antiVEGF via Akouos Delivery Device

• Registration Number: NCT06517888
• Lead Sponsor: Akouos, Inc.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-07-19
• Study First Submitted QC: 2024-07-19
• Study First Posted: 2024-07-24
• Status Verified: 2024-08
• Study Start: 2025-01-17
• Last Update Submitted: 2025-03-10
• Last Update Posted: 2025-03-12
• Primary Completion: 2029-08
• Study Completion: 2029-08

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 27

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1	AAVAnc80-antiVEGF via Akouos Delivery Device	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.
Cohort 3	AAVAnc80-antiVEGF via Akouos Delivery Device	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
Cohort 2	AAVAnc80-antiVEGF via Akouos Delivery Device	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.

Primary Outcome Measures

Name	Time	Method
Frequency of Adverse Events (AEs)	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 Outcome Measures

Name	Time	Method
Performance of Akouos Delivery Device	through trial completion, approximately one year	Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-antiVEGF to the intracochlear space
Tumor Volume	through trial completion, approximately one year	Change from baseline in tumor size via MRI (volumetric analysis)

Trial Locations

Locations (3)

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

Vanderbilt Bill Wilkerson Center; 🇺🇸 Nashville, Tennessee, United States

University of Texas Southwestern; 🇺🇸 Dallas, Texas, United States