Gene therapy trial aims to slow genetic form of dementia
NCT ID NCT04747431
First seen Nov 01, 2025 · Last updated Jun 13, 2026 · Updated 30 times
Summary
This study tests a single dose of PBFT02 gene therapy in 30 adults with frontotemporal dementia (FTD) caused by GRN or C9ORF72 gene mutations. The therapy delivers a working copy of the GRN gene to the brain to boost a key protein. Researchers are checking safety and whether it changes protein levels in spinal fluid and blood.
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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
Conditions
Explore the condition pages connected to this study.