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)

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

• Ages 30 to 75 years
• All sexes
• No healthy volunteers
• Adult, Older adult

Trial locations and contacts

19 sites in 9 countries. Contact the study team or a site near you to ask about taking part.

About this study

Official title: A Phase 1/2 Open-Label, Ascending Dose, Multicenter Study to Evaluate the Safety and Preliminary Efficacy of AVB-101 Administered by Bilateral Intrathalamic Infusion in Subjects With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)

Conditions

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

Being tested

Intrathalamic AAV.PGRN administration, Intrathalamic AVB-101

Keywords

Gene Therapy, AAV, Intrathalamic, Intraparenchymal, Progranulin, Behavioral Variant FTD, Primary Progressive Aphasia, PGRN, Granulin, Dementia, Dementia Gene Therapy, AAV9

Medical subject headings

Frontotemporal Dementia, Aphasia, Primary Progressive, Dementia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

• Intrathalamic AAV.PGRN administrationProcedure or surgery

  One-time MRI-guided stereotaxic infusion of AAV.PGRN into the brain

• Intrathalamic AVB-101Genetic

  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.

What is being measured

The primary outcome is the main question the study is designed to answer.

1. Number and incidence of AEs and SAEs

   Type and incidence of adverse events

   Measured: Up to week 26

2. Change from baseline in the Mini-Mental State Examination (MMSE)

   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.

   Measured: Up to week 12

3. Incidence of treatment emergent suicidal ideation or behavior

   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.

   Measured: 26 week initial, 5-year total follow-up period

4. Incidence of treatment-emergent clinically significant abnormalities in clinical examination findings

   Measured: 5-year total follow-up period

5. Incidence of treatment-emergent clinically significant abnormalities in safety laboratory values

   Measured: 5-year total follow-up period

6. Change from baseline in brain structure

   Assessed by presence of any clinically significant MRI findings at post treatment visits including brain swelling or bleeding

   Measured: 5-year total follow-up period

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1. First posted3 Oct 2023
2. Study start30 Aug 2023
3. Primary completion1 Apr 2027 (estimated)
4. Study completion21 Mar 2030 (estimated)

Resources

• Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
• Other trials for Frontotemporal DementiaStudies of the same condition, open and under way.
• What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.