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Hope for genetic dementia: AL001 trial ends early

NCT ID NCT04374136

Disease control Sponsor: Alector Inc. Source: ClinicalTrials.gov ↗

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
The trial has finished. Results may not be published yet.
Stopped early This study
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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 study tested a drug called AL001 in 119 people who carry a mutation in the progranulin gene, putting them at high risk for or already diagnosed with frontotemporal dementia. The goal was to see if AL001 could slow the worsening of memory, behavior, and language problems. The trial was terminated early, so results are limited.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
AL001 (a drug given by IV infusion)
What this could lead to
If it worked, AL001 could slow or stop the progression of frontotemporal dementia in people with a specific genetic mutation.
What could go wrong
The trial was terminated early, so we may not get clear answers. It was also a small study (119 people) and only for a rare genetic form of the disease.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

119 people

The number who actually took part.

Started

Jul 2020

Finished

Jan 2026

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

25 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: * Persons with a progranulin gene mutation and at risk of developing FTD symptoms as evidenced by a biomarker, or persons with a progranulin gene mutation and diagnosed with FTD. * If symptomatic, one or more of the criteria for the diagnosis of possible behavioral variant FTD, or a diagnosis of Primary Progressive Aphasia. * Study partner who consents to study participation and who cares for/visits the participant daily for at least 5 hours per week. * Written informed consent must be obtained and documented (from the participant or, where jurisdictions allow it, from their legal decision maker). Exclusion Criteria: * Dementia due to a condition other than FTD including, but not limited to, Alzheimer's disease, Parkinson's disease, dementia with Lewy bodies, Huntington disease, or vascular dementia. * Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins. * Current uncontrolled hypertension, diabetes mellitus or thyroid disease. Clinically significant heart disease, liver disease or kidney disease. History or evidence of clinically significant brain disease other than FTD. * Females who are pregnant or breastfeeding, or planning to conceive within the study period. * Any experimental vaccine or gene therapy. * History of cancer within the last 5 years. * Current use of anticoagulant medications (e.g., coumadin, heparinoids, apixaban). * Residence in a skilled nursing facility, convalescent home, or long term care facility at screening; or requires continuous nursing care.

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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.

Frontotemporal dementia

Contacts and locations

Locations

  • ASST degli Spedali Civili di Brescia - Spedali Civili di Brescia

    Brescia, Italy

  • Box Hill Hospital

    Box Hill, 3128, Australia

  • CHRU Lille

    Lille, France

  • CHU de Bordeaux

    Bordeaux, France

  • Centro Hospitalar E Universitário de Coimbra EPE

    Coimbra, Portugal

  • Centro Hospitalar de Lisboa Norte, EPE - Hospital de Santa Maria

    Lisbon, 1649-035, Portugal

  • Centro Hospitalar do Porto - Hospital de Santo António

    Porto, 4099-001, Portugal

  • Dignity Health

    Phoenix, Arizona, 85013, United States

  • Eginitio University General Hospital of Athens - 1st University Neurology Clinic

    Athens, Attica, 115 28, Greece

  • Emory University

    Atlanta, Georgia, 30329, United States

  • Erasmus MC

    Rotterdam, Netherlands

  • Felix Platter Spital

    Basel, Switzerland

  • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

    Milan, Italy

  • Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta

    Milan, Italy

  • Fundación Para La Lucha Contra Las Enfermedades Neurológicas de La Infancia

    Buenos Aires, 2325, Argentina

  • Groupe Hospitalier Pitié Salpétrière

    Paris, France

  • Hospital CUF Descobertas

    Lisbon, Portugal

  • Hospital Clinic de Barcelona

    Barcelona, Spain

  • Hospital Universitario de Donostia

    Donostia / San Sebastian, Spain

  • Houston Methodist Institute for Academic Medicine

    Houston, Texas, 77030, United States

  • IRCCS - Centro S. Giovanni di Dio Fatebenefratelli

    Brescia, Italy

  • Indiana University Health Neuroscience Center

    Indianapolis, Indiana, 46202, United States

  • Irving Institute for Clinical and Translational Research

    New York, New York, 10032, United States

  • Istanbul University Medical Faculty

    Istanbul, Fatih, 34093, Turkey (Türkiye)

  • Johns Hopkins University School of Medicine

    Baltimore, Maryland, 21205, United States

  • Karolinska Universitetssjukhuset Huddinge - PPDS

    Huddinge, Sweden

  • Mayo Comprehensive Cancer Center - PPDS

    Rochester, Minnesota, 55905, United States

  • Nuovo Ospedale Civile S. Agostino-Estense di Baggiovara

    Baggiovara, Italy

  • Oregon Health and Science University

    Portland, Oregon, 97239, United States

  • PIA Fondazione Panico

    Tricase, Italy

  • Sunnybrook Research Institute - University of Toronto

    Toronto, Canada

  • The Queen Elizabeth Hospital

    Woodville, Australia

  • The University of Western Ontario

    London, Canada

  • UZ Leuven

    Leuven, Vlaams Brabant, 3000, Belgium

  • Uniklinik Köln

    Cologne, 50937, Germany

  • University College London

    London, United Kingdom

  • University General Hospital of Alexandroupolis - Department of Neurology

    Alexandroupoli, Evros, 68100, Greece

  • University Of Cincinnati Gardner Neuroscience institute

    Cincinnati, Ohio, 45219, United States

  • University of California San Diego

    La Jolla, California, 92093-0648, United States

  • University of Colorado

    Aurora, Colorado, 80045, United States

  • University of Kansas Alzheimer's Disease Center

    Fairway, Kansas, 66205, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • Universitätsklinikum Ulm

    Ulm, Germany

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.