Gene therapy trial targets inherited dementia
NCT ID NCT04408625
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a single-dose gene therapy called LY3884963 in 35 people with frontotemporal dementia caused by GRN gene mutations. The therapy aims to raise levels of a missing protein (progranulin) in the brain. Participants receive the treatment via injection into the fluid around the brain and are followed for 5 years to check safety and effects on dementia symptoms.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
LY3884963 (a gene therapy)
What this could lead to
If it works, this could slow or stop the progression of frontotemporal dementia in people with GRN mutations by restoring a missing protein.
What could go wrong
This is an early, first-in-human trial with only 35 participants, so safety and effectiveness are not yet proven. The treatment requires strong immune-suppressing drugs, which carry their own risks.
Disclaimer
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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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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