Could a single gene injection slow frontotemporal dementia?
NCT ID NCT04747431
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a gene therapy called PBFT02 for frontotemporal dementia (FTD) in people with specific genetic mutations. The therapy delivers a working copy of the GRN gene directly into the brain. The study involves 30 adults and primarily checks for safety and side effects, while also measuring changes in key proteins linked to the disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- PBFT02 (a gene therapy that delivers a working copy of the GRN gene into brain cells)
- What this could lead to
- If it works, this could point toward a treatment that slows or stops the progression of frontotemporal dementia in people with specific genetic mutations.
- What could go wrong
- This is a very early, small trial (30 people) focused on safety. Gene therapies can have serious side effects, and it is unknown if PBFT02 will improve symptoms or long-term outcomes.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Centro Hospitalar e Universitário de Coimbra
Coimbra, Portugal
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Eastern Health-Box Hill Hospital
Melbourne, Victoria, 3128, Australia
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Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)
São Paulo, Brazil
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Hospital das Clinicas da Universidade Federal de Minas Gerais (UFMG)
Minas Gerais, Brazil
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Michigan Alzheimer's Disease Center
Ann Arbor, Michigan, 48105, United States
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Montreal Neurological Institute-Hospital
Montreal, Quebec, H3A 2B4, Canada
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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University of Texas at Houston
Houston, Texas, 77030, United States
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University of Toronto, Toronto Western Hospital
Toronto, Ontario, M5T 2S8, Canada
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Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
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