Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma

Akouos

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Vestibular Schwannoma

Treatments: Combination Product: AAVAnc80-antiVEGF via Akouos Delivery Device

Study type: Interventional

Funder types: Industry

Identifiers

NCT06517888

AK-antiVEGF-101

Details and patient eligibility

About

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.

Enrollment: 27 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and 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 design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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27 participants in 3 patient groups

Cohort 1

Experimental group

Description:

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.

Treatment:

Combination Product: AAVAnc80-antiVEGF via Akouos Delivery Device

Cohort 2

Experimental group

Description:

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.

Treatment:

Combination Product: AAVAnc80-antiVEGF via Akouos Delivery Device

Cohort 3

Experimental group

Description:

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

Treatment:

Combination Product: AAVAnc80-antiVEGF via Akouos Delivery Device