New gene therapy targets rare dementia at its genetic root
NCT ID NCT04408625
First seen Jan 09, 2026 · Last updated May 21, 2026 · Updated 20 times
Summary
This study tests a single-dose gene therapy (LY3884963) in 35 adults with frontotemporal dementia caused by a progranulin gene mutation. The goal is to see if it safely raises progranulin levels in the body and brain, which may slow disease progression. Participants receive one of two doses and are followed for 5 years to monitor safety and effects on thinking and daily function.
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the original study
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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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AP-HM Hôpital de La Timone
Saint-Pierre, Marseille, 13386, France
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Centre Mémoire de Ressources
Lille, 59000, France
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Hospital Clinic de Barcelona, Villaroel 170 Servicio de Neurología
Barcelona, 08036, Spain
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Hospital Universitario de Donostia, Servicio De Neurologia, Consultas Externas Neurologia, San Sebastian, Guipúzcoa
Donostia / San Sebastian, 20014, Spain
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Hospital of the University of Pennsylvania, 3 West Gates Building, 3400 Spruce Street
Philadelphia, Pennsylvania, 19104, United States
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Lahey Hospital & Medical Center, 41 Burlington Mall Road
Burlington, Massachusetts, 01805, United States
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Le Ber, Institut du Cerveau et de la Moelle Epinière
Paris, 75013, France
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PPD Phase 1 Clinic, 100 West Gore Street, Suite 202
Orlando, Florida, 32806, United States
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Royal Prince Alfred Hospital, Brain & Mind Research Institute, 94 Mallet Street
Camperdown, New South Wales, 2050, Australia
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UZ Leuven, Neurologie Herestraat 49
Leuven, 3000, Belgium
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University College London,Queen Square, Dementia Research Building, London,
London, WC1N 3BG, United Kingdom
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k2 Medical Research-Maitland
Maitland, Florida, 32751-5669, United States
Conditions
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