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NCT Number: NCT03987295

A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)

A Phase 2 open label study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia.

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Key information

Conditions

Frontotemporal Dementia Brain Diseases Central Nervous System Diseases Dementia Frontotemporal Lobar Degeneration Mental Disorders Metabolic Diseases Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies TDP-43 Proteinopathies

Age range

18 year–85 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Lawson Health Research Institute, St. Joseph's, London, Ontario, Canada

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About this study

This is a Phase 2, multicenter, open label study evaluating the safety, tolerability, PK and PD of AL001 administered intravenously in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

AL001

Drug

60 mg/kg of AL001 every 4 weeks

Primary outcomes

  1. Severity of TEAEs

    Time frame: 197 weeks

    Number of TEAEs categorized by severity

  2. Severity of Treatment-Related TEAEs

    Time frame: 197 weeks

    Number of treatment-related TEAEs categorized by severity

  3. Any TEAE Leading to Study Drug Discontinuation

    Time frame: 197 weeks

    Number of TEAEs leading to study drug discontinuation

  4. Immunogenicity Antidrug Antibodies (ADA) Titer

    Time frame: 97 weeks

    Titer values of antidrug antibodies (ADAs) in participants receiving latozinemab at week 97/Part 1 end of study

  5. Confirmatory Immunogenicity Antidrug Antibodies (ADA) Responses

    Time frame: 97 weeks

    Presence of confirmatory antidrug antibodies (ADAs) in participants who test positive for ADA at week 97 (Part 1 End of Study).

  6. Change From Baseline in Sheehan Suicidality Tracking Scale

    Time frame: 197 weeks

    The Sheehan Suicidality Tracking Scale (S-STS) is a structured assessment tool used to evaluate the presence, severity, and frequency of suicidal ideation and behavior. It includes items that address passive thoughts of death, active suicidal ideation, intent, planning, suicide attempts, and non-suicidal self-injury. The S-STS total score ranges from 0 to 64, based on 16 items each rated from 0 (not at all) to 4 (extremely), with higher scores indicating greater severity of suicidal ideation, intent, or behavior. The total score provides a quantitative measure of suicidality severity and is sensitive to change over time, making it suitable for clinical monitoring and research use.

Secondary outcomes

  1. Longitudinal Percent Change From Baseline of CSF PGRN

    Time frame: 97 weeks

    The percent change from baseline to specified timepoints of PGRN in CSF. The baseline visit is labeled as week 1 and therefore the 96th week is labeled as week 97.

  2. Longitudinal Percent Change From Baseline in Plasma PGRN

    Time frame: 97 weeks

    The percent change from baseline to specified timepoints for PGRN in plasma. The baseline visit is labeled as week 1 and therefore the 96th week is labeled as week 97.

  3. Longitudinal Levels of Sortilin in WBCs

    Time frame: 97 weeks

    The overall change from baseline in Sortilin in WBCs.

  4. Latozinemab Concentration in Serum

    Time frame: 97 weeks

    Serum concentration of Latozinemab at week 97.

  5. Cmax of Latozinemab at Specified Timepoints

    Time frame: 97 weeks

    Maximum observed concentration of Latozinemab at week 96 of treatment.

  6. Ctrough of Latozinemab at Specified Timepoints

    Time frame: 97 weeks

    Trough concentration of Latozinemab at week 96 of treatment

  7. ARCmax of Latozinemab

    Time frame: 61 weeks

    Ratio of latozinemab Cmax at week 61 to the Cmax at week 1

Sponsors and collaborators

Lead sponsor

Alector Inc.

Industry

Collaborators

  • GlaxoSmithKline

Registry information

Official study title

A Phase 2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in Heterozygous Carriers of Granulin or C9orf72 Mutations Causative of Frontotemporal Dementia

Important dates

Study start
2019
Primary completion
2024
Study completion
2024
First posted
Jun 17, 2019
Registry last updated
Dec 5, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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