New drug targets genetic dementia in small trial
NCT ID NCT03987295
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a drug called AL001 in 33 people who carry a gene mutation that causes frontotemporal dementia, a brain disease that affects personality and language. The goal was to see if the drug is safe and how it affects certain proteins in the body. The trial was open-label, meaning everyone knew they were getting the drug, and it focused on safety and biological changes rather than symptom improvement.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- AL001 (latozinemab)
- What this could lead to
- If successful, this could point toward a treatment that slows or stops frontotemporal dementia in people with specific genetic mutations.
- What could go wrong
- This is an early-phase, small study (33 people) focused on safety and biological markers, not on whether the drug improves symptoms. It may not lead to an effective treatment.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Brain Research Center - PPDS
Amsterdam, 1081GN, Netherlands
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Erasmus University Medical Center
Rotterdam, 3015 GD, Netherlands
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Lawson Health Research Institute, St. Joseph's
London, Ontario, N6A 4V2, Canada
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5, Canada
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Technical University of Munich
München, 81675, Germany
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The Glenn Biggs Institute for Alzheimer's and Neurodegenerative Diseases UT Health San Antonio
San Antonio, Texas, 78229, United States
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UCSF
San Francisco, California, 94158, United States
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University College London
London, WC1N 3BG, United Kingdom
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University of Brescia
Brescia, 25123, Italy
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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University of Ulm
Ulm, 89081, Germany
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