Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma
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.
Condition
- Vestibular Schwannoma
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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.
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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 |
|
Recruiting Locations
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Akouos, Inc.