AL001 for Frontotemporal Dementia

No longer recruiting at 12 trial locations

Overseen By Study Lead

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Alector Inc.

Disqualifiers: Severe allergies, Substance abuse, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 24

Duration: 96 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called AL001 for individuals with frontotemporal dementia, a condition affecting behavior and language. The study aims to assess the safety of AL001, its tolerability, and its behavior in the body. Participants should have a specific genetic mutation linked to this type of dementia, such as the Granulin or C9orf72 mutation. This trial may suit those in good health who do not reside in long-term care facilities. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that AL001 is likely to be safe for humans?

A previous study found no major safety issues for people taking AL001 for frontotemporal dementia. This study focused on individuals with specific gene mutations related to the condition. Reports indicate that the treatment is generally well-tolerated, with most participants not experiencing serious side effects. While researchers continue to examine the safety data, early results appear promising. Those considering joining a trial for AL001 might find these findings helpful in understanding the treatment's safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike standard treatments for frontotemporal dementia, which often focus on managing symptoms, AL001 targets the underlying cause by addressing genetic mutations. Specifically, AL001 works by increasing levels of a protein called progranulin, which is crucial for brain health and is often deficient in people with this condition. This treatment is administered intravenously every four weeks, offering a new approach that could potentially slow down or alter the progression of the disease. Researchers are excited because AL001's unique mechanism of action could provide a more effective and long-lasting solution compared to current symptomatic treatments.

What evidence suggests that AL001 might be an effective treatment for frontotemporal dementia?

Research shows that AL001, which participants in this trial will receive, may help treat frontotemporal dementia, especially in individuals with specific genetic changes like GRN and C9orf72. Studies have found that AL001 can raise levels of PGRN, a protein that protects brain cells, potentially slowing the disease. Early results from past studies suggest that AL001 was generally safe and well-tolerated by participants. These findings offer hope that AL001 could help manage symptoms of frontotemporal dementia. ^{1 2 4 6 7}

Who Is on the Research Team?

Peter Ljubenkov, MD

Principal Investigator

University of California, San Francisco

Are You a Good Fit for This Trial?

This trial is for individuals in good physical health who have specific genetic mutations (GRN or C9orf72) linked to frontotemporal dementia. Women must not be pregnant or breastfeeding. Excluded are those living in long-term care facilities, with a history of substance abuse, or severe allergies to certain antibodies.

Inclusion Criteria

I am in good health with no significant medical issues found in recent exams or tests.

I carry a specific genetic mutation linked to certain neurological conditions.

I am not pregnant or breastfeeding.

Exclusion Criteria

You have a history of severe allergic reactions to certain types of medications made from antibodies or fusion proteins.

You have had problems with drinking too much alcohol or using drugs.

Participant resides in a skilled nursing facility, convalescent home, or long term care facility

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intravenous administration of AL001 every 4 weeks

96 weeks

24 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term to assess safety and tolerability

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• AL001

Trial Overview

The study tests the safety and effects of AL001 on patients with frontotemporal dementia due to GRN or C9orf72 mutations. It's an open-label Phase 2 trial, meaning everyone gets the drug and both researchers and participants know what's being administered.

How Is the Trial Designed?

Group I: Granulin and C9orf72 Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Alector Inc.

Lead Sponsor

Trials

11

Recruited

1,300+

GlaxoSmithKline

Industry Sponsor

Trials

4,834

Recruited

8,389,000+

Headquarters

London, UK

Known For

Vaccines & Medicines

Top Products

**Advair (salmeterol, fluticasone propionate)**, **Shingrix (shingles vaccine)**, **Augmentin (amoxicillin/clavulanate potassium)**, **Ventolin (salbutamol sulfate)

Dame Emma Walmsley

GlaxoSmithKline

Chief Executive Officer since 2017

MA in Classics and Modern Languages from Oxford University

Dr. Hal Barron

GlaxoSmithKline

Chief Medical Officer since 2018

MD from Harvard Medical School

Citations

1.

investors.alector.com

investors.alector.com/news-releases/news-release-details/alector-announces-topline-results-latozinemab-phase-3-trial

Press Release

Alector Announces Topline Results from Latozinemab Phase 3 Trial in Individuals with Frontotemporal Dementia Due to a GRN Mutation and Provides ...

2.

theaftd.org

theaftd.org/posts/front-page/alector-therapeutics-announced-results-from-their-phase-3-clinical-trial-evaluating-latozinemab/

Alector Therapeutics Announces Results From Their Phase ...

On October 21, 2025, Alector Therapeutics announced results from their Phase 3 INFRONT-3 clinical trial evaluating latozinemab (AL001) for ...

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04374136

A Phase 3 Study to Evaluate Efficacy and Safety of AL001 ...

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

4.

neurology.org

neurology.org/doi/10.1212/WNL.98.18_supplement.3173

A Phase 2 Study of AL001 in Frontotemporal Dementia ...

Conclusions: AL001 is being developed for the treatment of FTD-GRN to reduce the rate of neurodegeneration by increasing levels of PGRN and ...

5.

alz-journals.onlinelibrary.wiley.com

alz-journals.onlinelibrary.wiley.com/doi/abs/10.1002/alz.054593

Six months interim analysis of the phase 2 study of AL001 in ...

Preliminary results demonstrated that AL001 was generally safe and well tolerated in FTD-GRN participants in INFRONT-2. Chronic dosing led to a ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT03987295

NCT03987295 | A Phase 2 Study to Evaluate Safety of ...

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

7.

mayo.edu

mayo.edu/research/clinical-trials/diseases-conditions/frontotemporal-dementia

Frontotemporal Dementia Clinical Trials

The primary purpose of this study is to evaluate the efficacy of AL001 compared with placebo in carriers of progranulin gene (GRN) mutations causative of ...