New gene therapy targets inherited dementia in early trial
NCT ID NCT04747431
First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 27 times
Summary
This study tests a single dose of a gene therapy called PBFT02 in 30 adults with frontotemporal dementia caused by specific gene mutations. The treatment aims to deliver a working copy of the GRN gene to the brain to help control the disease. Researchers are primarily checking safety and how the therapy affects protein levels in spinal fluid and blood.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for FRONTOTEMPORAL DEMENTIA are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
Centro Hospitalar e Universitário de Coimbra
Coimbra, Portugal
-
Eastern Health-Box Hill Hospital
Melbourne, Victoria, 3128, Australia
-
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)
São Paulo, Brazil
-
Hospital das Clinicas da Universidade Federal de Minas Gerais (UFMG)
Minas Gerais, Brazil
-
Michigan Alzheimer's Disease Center
Ann Arbor, Michigan, 48105, United States
-
Montreal Neurological Institute-Hospital
Montreal, Quebec, H3A 2B4, Canada
-
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
-
University of Texas at Houston
Houston, Texas, 77030, United States
-
University of Toronto, Toronto Western Hospital
Toronto, Ontario, M5T 2S8, Canada
-
Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
Conditions
Explore the condition pages connected to this study.