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)
NCT ID: NCT06064890
Last Updated: 2025-12-17
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
9 participants
INTERVENTIONAL
2023-08-30
2030-10-31
Brief Summary
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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.
Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Cohort 1 (dose 1)
Initial dose, delivered as a one-time only, intrathalamic administration.
Intrathalamic AAV.PGRN administration
One-time MRI-guided stereotaxic infusion of AAV.PGRN into the brain
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.
Cohort 2 (dose 2)
Escalated dose, delivered as a one-time only, intrathalamic administration.
Intrathalamic AAV.PGRN administration
One-time MRI-guided stereotaxic infusion of AAV.PGRN into the brain
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.
Interventions
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Intrathalamic AAV.PGRN administration
One-time MRI-guided stereotaxic infusion of AAV.PGRN into the brain
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.
Eligibility Criteria
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Inclusion Criteria
* 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
* Any concurrent disease that may cause cognitive impairment unrelated to mutations in the GRN gene, such as other causes of dementia, neurosyphilis, hydrocephalus, stroke, small vessel ischemic disease, uncontrolled hypothyroidism, or vitamin B12 deficiency
* Clinically significant abnormality on MRI at Screening considered to be a contraindication to Intrathalamic infusion
* Surgically significant pattern of brain atrophy on MRI at Screening that interferes with planned neurosurgical trajectory
* Previous treatment with any gene or cell therapy
* Previous treatment with any investigational medicinal product (IMP) within 60 days or 5 half-lives (whichever is longer) prior to study drug treatment
* Concomitant disease, any clinically significant laboratory abnormality, or treatment which, in the opinion of the Investigator, may pose an unacceptable safety risk to the participant or interfere with study conduct or the participant's ability to comply with study procedures including neurosurgical administration under anesthesia
30 Years
75 Years
ALL
No
Sponsors
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AviadoBio Ltd
INDUSTRY
Responsible Party
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Locations
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The Ohio State University (OSU) Wexner Medical Center
Columbus, Ohio, United States
Vanderbilt University Medical Centre
Nashville, Tennessee, United States
Houston Methodist Hospital
Houston, Texas, United States
Sunnybrook Health Sciences Centre
Toronto, Ontario, Canada
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, , Italy
Amsterdam UMC
Amsterdam, , Netherlands
NEURO-CARE Sp. z o.o. Sp. Komandytowa
Katowice, , Poland
Neurologia Slaska Centrum Medyczne
Katowice, , Poland
Uniwersyteckie Centrum Kliniczne, SUM w Katowicach
Katowice, , Poland
Euromedis Sp. z o.o.
Szczecin, , Poland
Centrum Medyczne NeuroProtect Sp z o.o.
Warsaw, , Poland
Mazowiecki Szpital Brodnowski Sp. z o. o.
Warsaw, , Poland
Hospital Clinic Barcelona
Barcelona, , Spain
Hospital Universitari i Politecnic La Fe
Valencia, , Spain
Skåne University Hospital
Lund, , Sweden
Cambridge University Hospitals NHS Foundation Trust
Cambridge, , United Kingdom
University Hospital of Wales
Cardiff, , United Kingdom
University College London Hospitals
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Other Identifiers
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2022-002568-62
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
AVB-PGRN-001
Identifier Type: -
Identifier Source: org_study_id