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Tests treatment safety and results for Frontotemporal Dementia
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)
ClinicalTrials.gov ID: NCT06064890
What this study is testing
What is Intrathalamic AVB-101?
Intrathalamic AVB-101 is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for frontotemporal dementia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 30 to 75
You may be able to join if
- Male or female, 30 to 75 years of age
- 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
- Able and willing to comply with all procedures and the study visit schedule
You likely can't join if
- Severe dementia, defined as CDR + NACC FTLD global score of 3.0, or other symptoms that preclude the ability to comply with study procedures and/or...
- Any concurrent disease that may cause cognitive impairment unrelated to mutations in the GRN gene, such as other causes of dementia, neurosyphilis...
- 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
See the full eligibility criteria
- Male or female, 30 to 75 years of age
- 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
- 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...
- 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
- Severe dementia, defined as CDR + NACC FTLD global score of 3.0, or other symptoms that preclude the ability to comply with study procedures and/or pose unacceptable safety risk to the subject
- 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...
- 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 study team makes the final eligibility decision.
Where it's taking place
- Columbus, Ohio, United States
- Nashville, Tennessee, United States
- Houston, Texas, United States
- Leuven, Belgium
- Toronto, Ontario, Canada
- Milan, Italy
- Amsterdam, Netherlands
- Katowice, Poland
- Szczecin, Poland
- Warsaw, Poland
- Barcelona, Spain
- Valencia, Spain
- Lund, Sweden
- Cambridge, United Kingdom
- Cardiff, United Kingdom
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 30 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Columbus, Ohio, United States; Nashville, Tennessee, United States; Houston, Texas, United States; Leuven, Belgium; Toronto, Ontario, Canada; Milan, Italy and 10 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.