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

January 20, 2026 updated by: Alector Inc.

A Phase 3, Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
119

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Argentina
      • Buenos Aires, Argentina, 2325
        • Fundación para la lucha contra las enfermedades neurológicas de la infancia
  • Australia
      • Box Hill, Australia, 3128
        • Box Hill Hospital
      • Woodville, Australia
        • The Queen Elizabeth Hospital
  • Belgium
    • Vlaams Brabant
      • Leuven, Vlaams Brabant, Belgium, 3000
        • UZ Leuven
  • Canada
      • London, Canada
        • The University of Western Ontario
      • Toronto, Canada
        • Sunnybrook Research Institute - University of Toronto
  • France
      • Bordeaux, France
        • CHU de Bordeaux
      • Lille, France
        • CHRU Lille
      • Paris, France
        • Groupe Hospitalier Pitié Salpêtrière
  • Germany
      • Cologne, Germany, 50937
        • Uniklinik Koln
      • Ulm, Germany
        • Universitatsklinikum Ulm
  • Greece
    • Attica
      • Athens, Attica, Greece, 115 28
        • Eginitio University General Hospital of Athens - 1st University Neurology Clinic
    • Evros
      • Alexandroupoli, Evros, Greece, 68100
        • University General Hospital of Alexandroupolis - Department of Neurology
  • Italy
      • Baggiovara, Italy
        • Nuovo Ospedale Civile S. Agostino-Estense di Baggiovara
      • Brescia, Italy
        • IRCCS - Centro S. Giovanni di Dio Fatebenefratelli
      • Brescia, Italy
        • ASST degli Spedali Civili di Brescia - Spedali Civili di Brescia
      • Milan, Italy
        • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
      • Milan, Italy
        • Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta
      • Tricase, Italy
        • Pia Fondazione Panico
  • Netherlands
      • Rotterdam, Netherlands
        • Erasmus MC
  • Portugal
      • Coimbra, Portugal
        • Centro Hospitalar E Universitario de Coimbra EPE
      • Lisbon, Portugal
        • Hospital CUF Descobertas
      • Lisbon, Portugal, 1649-035
        • Centro Hospitalar de Lisboa Norte, EPE - Hospital de Santa Maria
      • Porto, Portugal, 4099-001
        • Centro Hospitalar do Porto - Hospital de Santo António
  • Spain
      • Barcelona, Spain
        • Hospital Clinic de Barcelona
      • Donostia / San Sebastian, Spain
        • Hospital Universitario DE Donostia
  • Sweden
      • Huddinge, Sweden
        • Karolinska Universitetssjukhuset Huddinge - PPDS
  • Switzerland
      • Basel, Switzerland
        • Felix Platter Spital
  • Turkey (Türkiye)
    • Fatih
      • Istanbul, Fatih, Turkey (Türkiye), 34093
        • Istanbul University Medical Faculty
  • United Kingdom
      • London, United Kingdom
        • University College London
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • Dignity Health
    • California
      • La Jolla, California, United States, 92093-0648
        • University Of California San Diego
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Emory University
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University Health Neuroscience Center
    • Kansas
      • Fairway, Kansas, United States, 66205
        • University of Kansas Alzheimer's Disease Center
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Johns Hopkins University School of Medicine
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Comprehensive Cancer Center - PPDS
    • New York
      • New York, New York, United States, 10032
        • Irving Institute for Clinical and Translational Research
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • University of Cincinnati Gardner Neuroscience Institute
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center
    • Texas
      • Houston, Texas, United States, 77030
        • Houston Methodist Institute for Academic Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

25 years to 85 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: AL001
AL001 every 4 weeks
Drug: AL001
Administered via intravenous (IV) infusion
Placebo Comparator: Placebo
Placebo every 4 weeks
Drug: Placebo
Administered via intravenous (IV) infusion
Experimental: Open label - AL001
AL001 every 4 weeks
Drug: Open label - AL001
Administered via intravenous (IV) infusion

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Evaluation of efficacy of AL001 as measured by the CDR® plus NACC FTLD-SB
Time Frame: Through study completion, on average up to 96 weeks
The Clinical Dementia Rating Dementia Staging Instrument PLUS National Alzheimer's Disease Coordinating Center frontotemporal lobar degeneration Behavior & Language Domains Sum of Boxes (CDR® plus NACC FTLD-SB) is administered by a healthcare professional and based on individual ratings of the eight domains: memory, orientation, judgment and problem solving, community affairs, home and hobbies, personal care, language and behavior. Impairment is scored on a scale in which none = 0, questionable = 0.5, mild = 1, moderate = 2 and severe = 3. The 8 individual domain ratings, or "box scores", were added together to give the CDR® plus NACC FTLD-SB which ranges from 0-24. Higher score indicates severe impairment.
Through study completion, on average up to 96 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change in Clinical Global Impression-Severity (CGI-S) Score
Time Frame: Baseline to 96 weeks
The CGI-S is used by a clinician to rate the severity of a participant's disease relative to the clinician's past experience with patients who have the same disease using an ordinal scale ranging from 1=normal, not at all ill; 2=borderline ill; 3=mildly ill; 4=moderately ill; 5=markedly ill; 6=severely ill; and 7=among the most extremely ill patients. Higher scores indicate worsening.
Baseline to 96 weeks
Change in Clinical Global Impression-Improvement (CGI-I) Score
Time Frame: Baseline to 96 weeks
The CGI-I is used by a clinician to rate how much a participant's disease has improved or worsened relative to baseline using an ordinal scale ranging from 1=very much improved; 2=much improved; 3=minimally improved; 4=no change from baseline; 5=minimally worse; 6= much worse; and 7=very much worse. Higher scores indicate worsening.
Baseline to 96 weeks
Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Score
Time Frame: Baseline to 96 weeks
RBANS is 20 to 25 minute battery developed for cognitive assessment, detection, and characterization of dementia. RBANS includes 12 subtests that measure following 5 indices: (1)Attention Index, composed of Digit Span and Coding; (2)Language Index, consisting of Picture Naming and Semantic Fluency subtests; (3)Visuospatial/Construction Index, made up of Figure Copy and Line Orientation subtests; (4)Immediate Memory Index, composed of List Learning and Story Memory subtests, and (5)Delayed Memory Index, consisting of List Recall, List Recognition, Story Recall, and Figure Recall subtests. Completion of RBANS yields 5 index scores based on participant performance on various subtests, as well as a composite Total Index score for battery. Total index scores range from 40 to 160, and are normalized to a mean of 100 and standard deviation (SD) of 15. Higher scores indicate less impairment.
Baseline to 96 weeks
Pharmacodynamic Biomarkers
Time Frame: Baseline to 96 weeks
Change in magnetic resonance imaging and blood-based biomarkers and optional CSF biomarkers (neurofilament light chain and progranulin)
Baseline to 96 weeks
Evaluation of safety and tolerability of AL001: Incidence of adverse events
Time Frame: Baseline to 96 weeks
Incidence of adverse events
Baseline to 96 weeks

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Optional Open-Label Extension
Time Frame: 96 weeks
Assess the long-term safety and tolerability of AL001 in participants who have completed 96 week of treatment
96 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alector Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
GlaxoSmithKline

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: TBD TBD

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 23, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 1, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 6, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 23, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 30, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 5, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 21, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 20, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AL001-3

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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