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.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
33 people
The number who actually took part.
- Started
-
Sep 2019
- Finished
-
Jun 2024
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 85 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * At screening, female participants must be nonpregnant and nonlactating * In good physical health on the basis of no clinically significant findings from medical history, physical examinations (PEs), laboratory tests, ECGs, and vital signs. * Participant is a carrier of a loss of function progranulin gene (GRN) mutation or carrier of a hexanucleotide repeat expansion C9orf72 mutation Exclusion Criteria: * Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins. * History of alcohol abuse or substance abuse * Participant resides in a skilled nursing facility, convalescent home, or long term care facility
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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
-
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
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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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