Hope for genetic dementia: AL001 trial ends early

NCT ID NCT04374136

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

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.

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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

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