Gene therapy trial hopes to halt rare dementia
NCT ID NCT04747431
First seen Nov 01, 2025 · Last updated Jun 23, 2026 · Updated 32 times
Summary
This early-stage trial tests a gene therapy called PBFT02 in 30 adults with frontotemporal dementia caused by mutations in the GRN or C9ORF72 genes. The therapy delivers a working copy of the GRN gene directly into the brain to help restore a missing protein. The main goals are to check safety and measure changes in protein levels in spinal fluid and blood.
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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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
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 to the brain)
What this could lead to
If successful, 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 an early, small trial (30 people) focused on safety, so it may not show clear benefit. Gene therapies can have side effects like immune reactions or nerve damage.
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.