Actively Recruiting
A Phase 1/2 Study to Evaluate Safety and Preliminary Effects of AVB-101 Gene Therapy Delivered by Intrathalamic Infusion in Frontotemporal Dementia with Progranulin Mutations
Led by AviadoBio Ltd · Updated on 2026-04-13
18
Participants Needed
19
Research Sites
207 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying an investigational gene therapy called AVB-101 for people with Frontotemporal Dementia caused by Progranulin gene mutations (FTD-GRN). This form of dementia affects behavior, language, and movement due to low levels of the progranulin protein in the brain, leading to nerve cell death. The study aims to find out if a single treatment with AVB-101 is safe, can restore progranulin levels, and may slow or stop disease progression. Participants will receive a one-time infusion of AVB-101 directly into the brain using MRI-guided stereotaxic surgery. The study includes four groups receiving ascending doses of the gene therapy. There is no placebo group; everyone receives the active treatment. After the infusion, participants will be followed for up to 5 years to monitor safety and effects. During the study, participants will undergo regular assessments including cognitive tests like the Mini-Mental State Examination, brain imaging, and blood and cerebrospinal fluid tests to measure progranulin and other markers. Researchers will track any side effects, changes in brain structure, and clinical symptoms over the 5-year period. Participants will also have support from a study partner to help with assessments and study procedures throughout the trial.
CONDITIONS
Brief Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged 30 to 75 years
- Carriers of a pathogenic GRN mutation
- Diagnosis of Frontotemporal Dementia with a CDR + NACC FTLD global score of 0.5, 1.0, or 2.0
- Presence of one or more criteria for possible behavioral variant FTD or primary progressive aphasia
- Able and willing to comply with study procedures and visit schedule
- Able and willing to provide informed consent or have a legal representative provide consent if capacity is lost
- Has an identified, informed study partner willing to support and assess the participant during the study
You will not qualify if you...
- Severe dementia with a CDR + NACC FTLD global score of 3.0 or symptoms preventing study compliance or posing safety risks
- Any other disease causing cognitive impairment unrelated to GRN mutations, such as other dementias, neurosyphilis, hydrocephalus, stroke, small vessel ischemic disease, uncontrolled hypothyroidism, or vitamin B12 deficiency
- Clinically significant abnormality or brain atrophy on MRI that contraindicates intrathalamic infusion or neurosurgical approach
- Previous treatment with any gene or cell therapy
- Previous treatment with any investigational medicinal product within 60 days or 5 half-lives prior to study treatment
- Any clinically significant disease, laboratory abnormality, or treatment posing safety risks or interfering with study procedures including neurosurgery under anesthesia
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single administration with monitoring up to 26 weeks
Participants receive a one-time MRI-guided stereotaxic infusion of AVB-101 gene therapy into the brain.
Multiple follow-up visits during the first 26 weeks
Duration - Up to 5 years
Participants are monitored for safety, cognitive changes, and biological markers for up to 5 years after treatment.
Periodic visits over the 5-year follow-up period
Trial Site Locations
Total: 19 locations
1
The Ohio State University (OSU) Wexner Medical Center
Columbus, Ohio, United States, 43210
Actively Recruiting
2
Vanderbilt University Medical Centre
Nashville, Tennessee, United States, 37232
Actively Recruiting
3
Houston Methodist Hospital
Houston, Texas, United States, 77030
Actively Recruiting
4
UZ Leuven
Leuven, Belgium
Actively Recruiting
5
Sunnybrook Health Sciences Centre
Toronto, Ontario, Canada
Actively Recruiting
6
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Italy
Actively Recruiting
7
Amsterdam UMC
Amsterdam, Netherlands
Actively Recruiting
8
NEURO-CARE Sp. z o.o. Sp. Komandytowa
Katowice, Poland
Actively Recruiting
9
Neurologia Slaska Centrum Medyczne
Katowice, Poland
Actively Recruiting
10
Uniwersyteckie Centrum Kliniczne, SUM w Katowicach
Katowice, Poland
Actively Recruiting
11
Euromedis Sp. z o.o.
Szczecin, Poland, 70-111
Actively Recruiting
12
Centrum Medyczne NeuroProtect Sp z o.o.
Warsaw, Poland
Actively Recruiting
13
Mazowiecki Szpital Brodnowski Sp. z o. o.
Warsaw, Poland
Actively Recruiting
14
Hospital Clinic Barcelona
Barcelona, Spain, 08036
Actively Recruiting
15
Hospital Universitari i Politecnic La Fe
Valencia, Spain, 46026
Actively Recruiting
16
Skåne University Hospital
Lund, Sweden
Actively Recruiting
17
Cambridge University Hospitals NHS Foundation Trust
Cambridge, United Kingdom, CB2 0QQ
Actively Recruiting
18
University Hospital of Wales
Cardiff, United Kingdom, CF14 4XW
Actively Recruiting
19
University College London Hospitals
London, United Kingdom
Actively Recruiting
Research Team
A
AviadoBio Clinical Trials
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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