Gene therapy infused directly into brain aims to halt rare dementia
NCT ID NCT06064890
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a gene therapy called AVB-101 for people with frontotemporal dementia caused by GRN gene mutations. The therapy delivers a working copy of the GRN gene directly into the brain during a one-time procedure. The study will enroll 18 adults aged 30-75 to check safety and whether it can restore normal levels of a key protein, potentially slowing 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
AVB-101 (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 slow or stop the progression of a devastating early-onset dementia by restoring a missing protein in the brain.
What could go wrong
This is a very early, small trial (18 people) testing safety first. Gene therapy delivered directly to the brain carries risks like brain swelling or bleeding, and it may not improve symptoms.
Disclaimer
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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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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Amsterdam UMC
RECRUITINGAmsterdam, Netherlands
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Cambridge University Hospitals NHS Foundation Trust
RECRUITINGCambridge, CB2 0QQ, United Kingdom
Contact Email: •••••@•••••
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Centrum Medyczne NeuroProtect Sp z o.o.
RECRUITINGWarsaw, Poland
Contact Email: •••••@•••••
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Euromedis Sp. z o.o.
RECRUITINGSzczecin, 70-111, Poland
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Fondazione IRCCS Istituto Neurologico Carlo Besta
RECRUITINGMilan, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Clinic Barcelona
RECRUITINGBarcelona, 08036, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Universitari i Politecnic La Fe
RECRUITINGValencia, 46026, Spain
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Houston Methodist Hospital
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Mazowiecki Szpital Brodnowski Sp. z o. o.
RECRUITINGWarsaw, Poland
Contact Email: •••••@•••••
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NEURO-CARE Sp. z o.o. Sp. Komandytowa
RECRUITINGKatowice, Poland
Contact Email: •••••@•••••
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Neurologia Slaska Centrum Medyczne
RECRUITINGKatowice, Poland
Contact Email: •••••@•••••
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Skåne University Hospital
RECRUITINGLund, Sweden
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Sunnybrook Health Sciences Centre
RECRUITINGToronto, Ontario, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Ohio State University (OSU) Wexner Medical Center
RECRUITINGColumbus, Ohio, 43210, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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UZ Leuven
RECRUITINGLeuven, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University College London Hospitals
RECRUITINGLondon, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University Hospital of Wales
RECRUITINGCardiff, CF14 4XW, United Kingdom
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Uniwersyteckie Centrum Kliniczne, SUM w Katowicach
RECRUITINGKatowice, Poland
Contact Email: •••••@•••••
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Vanderbilt University Medical Centre
RECRUITINGNashville, Tennessee, 37232, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••