New drug shows promise for inherited dementia

NCT ID NCT03987295

First seen Jan 04, 2026 · Last updated May 14, 2026 · Updated 19 times

Summary

This study tested an experimental drug called AL001 in 33 people with frontotemporal dementia caused by specific gene mutations (GRN or C9orf72). The main goals were to check safety and how the drug moves through the body. Researchers also measured changes in certain proteins in spinal fluid and blood to see if the drug was working as expected.

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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

Conditions

Explore the condition pages connected to this study.