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

NCT ID: NCT03987295

Last Updated: 2025-12-05

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

33 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-09-27

Study Completion Date

2024-06-05

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Frontotemporal Dementia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Granulin and C9orf72

IV administration of AL001; 60 mg/kg, every 4 weeks \[q4w\]

Group Type EXPERIMENTAL

AL001

Intervention Type DRUG

60 mg/kg of AL001 every 4 weeks

Interventions

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AL001

60 mg/kg of AL001 every 4 weeks

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 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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Peter Ljubenkov, MD

Role: PRINCIPAL_INVESTIGATOR

University of California, San Francisco

Locations

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UCSF

San Francisco, California, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

The Glenn Biggs Institute for Alzheimer's and Neurodegenerative Diseases UT Health San Antonio

San Antonio, Texas, United States

Site Status

Lawson Health Research Institute, St. Joseph's

London, Ontario, Canada

Site Status

Sunnybrook Health Sciences Centre

Toronto, Ontario, Canada

Site Status

Technical University of Munich

München, , Germany

Site Status

University of Ulm

Ulm, , Germany

Site Status

University of Brescia

Brescia, , Italy

Site Status

Brain Research Center - PPDS

Amsterdam, , Netherlands

Site Status

Erasmus University Medical Center

Rotterdam, , Netherlands

Site Status

University College London

London, , United Kingdom

Site Status

Countries

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United States Canada Germany Italy Netherlands United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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AL001-2

Identifier Type: -

Identifier Source: org_study_id

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