Gene therapy trial targets inherited dementia

NCT ID NCT04408625

Ongoing Disease control Sponsor: Prevail Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jul 17, 2026 · Updated 3 times

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

frontotemporal dementia GRN-related frontotemporal lobar degeneration with Tdp43 inclusions

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

FRONTOTEMPORAL DEMENTIA

Contacts and locations

Locations

AP-HM Hôpital de La Timone

Saint-Pierre, Marseille, 13386, France
Centre Mémoire de Ressources

Lille, 59000, France
Hospital Clinic de Barcelona, Villaroel 170 Servicio de Neurología

Barcelona, 08036, Spain
Hospital Universitario de Donostia, Servicio De Neurologia, Consultas Externas Neurologia, San Sebastian, Guipúzcoa

Donostia / San Sebastian, 20014, Spain
Hospital of the University of Pennsylvania, 3 West Gates Building, 3400 Spruce Street

Philadelphia, Pennsylvania, 19104, United States
Lahey Hospital & Medical Center, 41 Burlington Mall Road

Burlington, Massachusetts, 01805, United States
Le Ber, Institut du Cerveau et de la Moelle Epinière

Paris, 75013, France
PPD Phase 1 Clinic, 100 West Gore Street, Suite 202

Orlando, Florida, 32806, United States
Royal Prince Alfred Hospital, Brain & Mind Research Institute, 94 Mallet Street

Camperdown, New South Wales, 2050, Australia
UZ Leuven, Neurologie Herestraat 49

Leuven, 3000, Belgium
University College London,Queen Square, Dementia Research Building, London,

London, WC1N 3BG, United Kingdom
k2 Medical Research-Maitland

Maitland, Florida, 32751-5669, United States

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