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New drug targets genetic dementia in small trial

NCT ID NCT03987295

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

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

  • 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

More trials for these conditions

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