Brain injection aims to halt devastating dementia
NCT ID NCT04747431
Summary
This early-stage study is testing a one-time gene therapy called PBFT02 for adults with specific genetic forms of frontotemporal dementia (FTD). The therapy is delivered via a single injection into the spinal fluid, aiming to deliver a working copy of a missing or faulty gene to the brain. The main goals are to check if the treatment is safe and to see if it can increase a key protein and potentially slow the disease's progression over a 5-year follow-up period.
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Contacts and locations
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Study contacts
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Centro Hospitalar e Universitário de Coimbra
RECRUITINGCoimbra, Portugal
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Eastern Health-Box Hill Hospital
RECRUITINGMelbourne, Victoria, 3128, Australia
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Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)
RECRUITINGSão Paulo, Brazil
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Hospital das Clinicas da Universidade Federal de Minas Gerais (UFMG)
RECRUITINGMinas Gerais, Brazil
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Michigan Alzheimer's Disease Center
RECRUITINGAnn Arbor, Michigan, 48105, United States
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Montreal Neurological Institute-Hospital
RECRUITINGMontreal, Quebec, H3A 2B4, Canada
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University of Pennsylvania
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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University of Texas at Houston
RECRUITINGHouston, Texas, 77030, United States
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University of Toronto, Toronto Western Hospital
RECRUITINGToronto, Ontario, M5T 2S8, Canada
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Vanderbilt University Medical Center
RECRUITINGNashville, Tennessee, 37232, United States
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Conditions
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