A Phase 3 Study to Evaluate Efficacy and Safety of AL001 in Frontotemporal Dementia (INFRONT-3)

NCT ID: NCT04374136

Last Updated: 2025-08-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

110 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-07-23

Study Completion Date

2027-08-03

Brief Summary

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A phase 3 double blind, placebo controlled study evaluating the efficacy and safety of AL001 in participants at risk for or with frontotemporal dementia due to heterozygous mutations in the progranulin gene.

Detailed Description

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This is a phase 3 double blind, placebo controlled study evaluating the efficacy and safety of AL001 administered intravenously in participants at risk for or with frontotemporal dementia due to heterozygous mutations in the progranulin gene. Study completion marks the end of the open label extension period following the 96-week blinded portion of the study.

Conditions

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Frontotemporal Dementia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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AL001

AL001 every 4 weeks

Group Type EXPERIMENTAL

AL001

Intervention Type DRUG

Administered via intravenous (IV) infusion

Placebo

Placebo every 4 weeks

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Administered via intravenous (IV) infusion

Open label - AL001

AL001 every 4 weeks

Group Type EXPERIMENTAL

Open label - AL001

Intervention Type DRUG

Administered via intravenous (IV) infusion

Interventions

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AL001

Administered via intravenous (IV) infusion

Intervention Type DRUG

Placebo

Administered via intravenous (IV) infusion

Intervention Type DRUG

Open label - AL001

Administered via intravenous (IV) infusion

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

25 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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GlaxoSmithKline

INDUSTRY

Sponsor Role collaborator

Alector Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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TBD TBD

Role: PRINCIPAL_INVESTIGATOR

Locations

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Dignity Health

Phoenix, Arizona, United States

Site Status

University of California San Diego

La Jolla, California, United States

Site Status

University of Colorado

Aurora, Colorado, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

Indiana University Health Neuroscience Center

Indianapolis, Indiana, United States

Site Status

University of Kansas Alzheimer's Disease Center

Fairway, Kansas, United States

Site Status

Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

Mayo Comprehensive Cancer Center - PPDS

Rochester, Minnesota, United States

Site Status

Irving Institute for Clinical and Translational Research

New York, New York, United States

Site Status

University Of Cincinnati Gardner Neuroscience institute

Cincinnati, Ohio, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Houston Methodist Institute for Academic Medicine

Houston, Texas, United States

Site Status

Fundación Para La Lucha Contra Las Enfermedades Neurológicas de La Infancia

Buenos Aires, , Argentina

Site Status

Box Hill Hospital

Box Hill, , Australia

Site Status

The Queen Elizabeth Hospital

Woodville, , Australia

Site Status

UZ Leuven

Leuven, Vlaams Brabant, Belgium

Site Status

The University of Western Ontario

London, , Canada

Site Status

Sunnybrook Research Institute - University of Toronto

Toronto, , Canada

Site Status

CHU de Bordeaux

Bordeaux, , France

Site Status

CHRU Lille

Lille, , France

Site Status

Groupe Hospitalier Pitié Salpétrière

Paris, , France

Site Status

Uniklinik Köln

Cologne, , Germany

Site Status

Universitätsklinikum Ulm

Ulm, , Germany

Site Status

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

Athens, Attica, Greece

Site Status

University General Hospital of Alexandroupolis - Department of Neurology

Alexandroupoli, Evros, Greece

Site Status

Nuovo Ospedale Civile S. Agostino-Estense di Baggiovara

Baggiovara, , Italy

Site Status

ASST degli Spedali Civili di Brescia - Spedali Civili di Brescia

Brescia, , Italy

Site Status

IRCCS - Centro S. Giovanni di Dio Fatebenefratelli

Brescia, , Italy

Site Status

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, , Italy

Site Status

Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta

Milan, , Italy

Site Status

PIA Fondazione Panico

Tricase, , Italy

Site Status

Erasmus MC

Rotterdam, , Netherlands

Site Status

Centro Hospitalar do Porto - Hospital de Santo António

Porto, Porto District, Portugal

Site Status

Centro Hospitalar E Universitário de Coimbra EPE

Coimbra, , Portugal

Site Status

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

Lisbon, , Portugal

Site Status

Hospital CUF Descobertas

Lisbon, , Portugal

Site Status

Hospital Clinic de Barcelona

Barcelona, , Spain

Site Status

Hospital Universitario de Donostia

Donostia / San Sebastian, , Spain

Site Status

Karolinska Universitetssjukhuset Huddinge - PPDS

Huddinge, , Sweden

Site Status

Felix Platter Spital

Basel, , Switzerland

Site Status

Istanbul University Medical Faculty

Istanbul, Fatih, Turkey (Türkiye)

Site Status

University College London

London, , United Kingdom

Site Status

Countries

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United States Argentina Australia Belgium Canada France Germany Greece Italy Netherlands Portugal Spain Sweden Switzerland Turkey (Türkiye) United Kingdom

Other Identifiers

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AL001-3

Identifier Type: -

Identifier Source: org_study_id

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