Experimental gene therapy targets genetic form of dementia
NCT ID NCT04747431
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 26 times
Summary
This study tests a one-time gene therapy called PBFT02 for frontotemporal dementia (FTD) in adults who have a mutation in the GRN or C9ORF72 gene. The therapy aims to deliver a working copy of the GRN gene to the brain to help control the disease. About 30 participants will receive a single dose to check safety and how well it works. This is an early-stage trial, so the main goal is to see if the treatment is safe and to measure changes in key proteins in the blood and spinal fluid.
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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.