A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN)

Part of paid clinical trials in Columbus, Ohio.

Sponsor
AviadoBio Ltd
Study ID
NCT06064890
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Dementia, Frontotemporal
  • FTD
  • FTD-GRN
  • Frontotemporal Dementia

Eligibility Criteria

Sex
ALL
Age
30 Years - 75 Years
Healthy Volunteers
Not accepted

Interventions

  • Intrathalamic AAV.PGRN administration — PROCEDURE
    One-time MRI-guided stereotaxic infusion of AAV.PGRN into the brain
  • Intrathalamic AVB-101 — GENETIC
    AVB-101 is made from an adeno-associated virus, serotype 9 (AAV9). AAVs are small viruses that are naturally occurring and do not cause illness or infection on their own. AVB-101 has been modified to contain a copy of the correct (non-mutated) GRN gene, plus some other genetic material to enable the GRN gene to function inside neurons (cells within the brain). AVB-101 has also been modified so that it cannot divide and make new copies of itself (known as 'replication'), which means that it cannot cause disease or a large immune response in your body.

Study Details

The goal of this clinical study is to learn about an investigational gene therapy product called AVB-101, which is designed to treat a disease called Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). FTD-GRN is an early-onset form of dementia, a progressive brain disorder that affects behavior, language and movement. These symptoms result from below normal levels of a protein called progranulin (PGRN) in the brain, which leads to the death of nerve cells (neurons), affecting the brain's ability to function. The main questions that the study aims to answer are: 1. Is a one-time treatment with AVB-101 safe for patients with FTD-GRN? 2. Does a one-time treatment with AVB-101 restore PGRN levels to at least normal levels? 3. Could AVB-101 work as a treatment to slow down or stop progression of FTD-GRN? In this study there is no placebo (a dummy pill or treatment used for comparison purposes), so all participants will receive a one-time treatment of AVB-101 delivered directly to the brain, with follow-up assessments for 5 years.

Key Dates

Start date
Aug 30, 2023
Status verified
Apr 2026
Primary completion
Apr 1, 2026
Completion
Mar 21, 2030

Study Design

Enrollment
18 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1 (dose 1)
    Initial dose
  • Experimental: Cohort 2 (dose 2)
    Escalated dose
  • Experimental: Cohort 3 (dose 3)
    Additional dose
  • Experimental: Cohort 4 (dose 4)
    Additional dose

Primary Outcome Measure

Number and incidence of AEs and SAEs [ Time Frame: Up to week 26 ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
The Ohio State University (OSU) Wexner Medical CenterColumbusOhio43210
Jennifer Icenhour
[email protected]
614-293-6882
Andrea Davis
[email protected]
614-688-6412
Vanderbilt University Medical CentreNashvilleTennessee37232
Jerica Reeder
[email protected]
615-875-2987
Houston Methodist HospitalHoustonTexas77030
Sophia Jung
[email protected]
346-238-2005

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The Ohio State University (OSU) Wexner Medical Center· Columbus, OHVanderbilt University Medical Centre· Nashville, TNHouston Methodist Hospital· Houston, TX

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