Actively 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)
Led by AviadoBio Ltd · Updated on 2026-04-13
18
Participants Needed
19
Research Sites
342 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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, 30 to 75 years of age
- Carriers of a pathogenic GRN mutation
- Frontotemporal Dementia with 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 all study procedures and visit schedule
- Able and willing to provide written informed consent and designate a legal representative if capacity is lost
- Have an identified, informed study partner able to support and provide assessments during the study
You will not qualify if you...
- Severe dementia with CDR + NACC FTLD global score of 3.0 or symptoms preventing compliance or posing safety risks
- Any other disease causing cognitive impairment unrelated to GRN mutations, including neurosyphilis, hydrocephalus, stroke, small vessel ischemic disease, uncontrolled hypothyroidism, or vitamin B12 deficiency
- Clinically significant abnormality or brain atrophy pattern on MRI that contraindicates intrathalamic infusion
- Previous treatment with any gene or cell therapy
- Previous investigational medicinal product treatment within 60 days or 5 half-lives before study drug
- Any disease, laboratory abnormality, or treatment posing unacceptable safety risk or interfering with study participation including neurosurgical procedures under anesthesia
AI-Screening
AI-Powered Screening
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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
CONTACT
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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