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Study identifier: NCT06064890 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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)

Condition: Frontotemporal Dementia · FTD · FTD-GRN  ·  Sponsor: AviadoBio Ltd

PhasePhase 1/Phase 2
Planned participants18
Who can joinAll sexes, 30 Years to 75 Years
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

AviadoBio Clinical Trials  ·  +44 203-089-7917  ·  clinicaltrials@aviadobio.com

Always discuss trial participation with your own doctor first.

Locations (19)

The Ohio State University (OSU) Wexner Medical CenterColumbus, Ohio, United StatesRecruiting
Vanderbilt University Medical CentreNashville, Tennessee, United StatesRecruiting
Houston Methodist HospitalHouston, Texas, United StatesRecruiting
UZ LeuvenLeuven, BelgiumRecruiting
Sunnybrook Health Sciences CentreToronto, Ontario, CanadaRecruiting
Fondazione IRCCS Istituto Neurologico Carlo BestaMilan, ItalyRecruiting
Amsterdam UMCAmsterdam, NetherlandsRecruiting
NEURO-CARE Sp. z o.o. Sp. KomandytowaKatowice, PolandRecruiting
Neurologia Slaska Centrum MedyczneKatowice, PolandRecruiting
Uniwersyteckie Centrum Kliniczne, SUM w KatowicachKatowice, PolandRecruiting
Euromedis Sp. z o.o.Szczecin, PolandRecruiting
Centrum Medyczne NeuroProtect Sp z o.o.Warsaw, PolandRecruiting

+ 7 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT06064890