NCT06064890 · AviadoBio Ltd
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)
(ASPIRE-FTD)
What this study is about
The goal of this clinical study is to learn about an experimental 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.
View original scientific description
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.
Interventions
PROCEDURE
Intrathalamic AAV.PGRN administration
One-time MRI-guided stereotaxic infusion of AAV.PGRN into the brain
GENETIC
Intrathalamic AVB-101
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.
Primary outcome measures
Number and incidence of AEs and SAEs
Time frame: Up to week 26
Type and incidence of adverse events
Change from baseline in the Mini-Mental State Examination (MMSE)
Time frame: Up to week 12
Mini-Mental State Examination (MMSE) is a global assessment of cognitive status. Score range 0-30; higher scores reflect better cognitive function. Change in MMSE score from baseline visit to post-treatment visit will be assessed.
Incidence of treatment emergent suicidal ideation or behavior
Time frame: 26 week initial, 5-year total follow-up period
The Columbia-Suicide Severity Rating Scale (C-SSRS) is an assessment tool that evaluates suicidal ideation and behavior. C-SSRS will be measured at each visit to assess for absence/presence of suicidal ideation and/or behavior.
Incidence of treatment-emergent clinically significant abnormalities in clinical examination findings
Time frame: 5-year total follow-up period
Incidence of treatment-emergent clinically significant abnormalities in safety laboratory values
Time frame: 5-year total follow-up period
Change from baseline in brain structure
Time frame: 5-year total follow-up period
Assessed by presence of any clinically significant MRI findings at post treatment visits including brain swelling or bleeding
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Male or female, 30 to 75 years of age
- Carriers of a pathogenic GRN mutation
- FTD as evidenced by CDR + NACC FTLD global score of 0.5, 1.0, or 2.0
- Presence of 1 or more of the criteria for diagnosis of possible bvFTD or PPA
- A protocol defined minimum thalamic volume on each side on Screening MRI
- Able and willing to comply with all procedures and the study visit schedule
- Able and willing to give written informed consent prior to study participation, and agree to designate a legal representative to act on their wishes to continue participation should they lose capacity to consent at some point during the study
- An identified, informed study partner who is able and willing to support the participant in the study and to provide assessments of the participant during the study
Exclusion criteria
- Severe dementia, defined as CDR + NACC FTLD global score of 3.0, or other symptoms that preclude the ability to comply with study procedures and/or pose unaccep
Where
- Columbus, Ohio
- Nashville, Tennessee
- Houston, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 13, 2026 · Source of record for eligibility and locations