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New drug targets genetic dementia in small trial

NCT ID NCT03987295

First seen Jan 04, 2026 · Last updated Jun 20, 2026 · Updated 25 times

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.

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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.

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Contacts and locations

Locations

  • Brain Research Center - PPDS

    Amsterdam, 1081GN, Netherlands

  • Erasmus University Medical Center

    Rotterdam, 3015 GD, Netherlands

  • Lawson Health Research Institute, St. Joseph's

    London, Ontario, N6A 4V2, Canada

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • Sunnybrook Health Sciences Centre

    Toronto, Ontario, M4N 3M5, Canada

  • Technical University of Munich

    München, 81675, Germany

  • The Glenn Biggs Institute for Alzheimer's and Neurodegenerative Diseases UT Health San Antonio

    San Antonio, Texas, 78229, United States

  • UCSF

    San Francisco, California, 94158, United States

  • University College London

    London, WC1N 3BG, United Kingdom

  • University of Brescia

    Brescia, 25123, Italy

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Ulm

    Ulm, 89081, Germany

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.

Conditions

The condition(s) this trial relates to.

frontotemporal dementia

As listed by the trial registrant

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