AL001 for Frontotemporal Dementia

No longer recruiting at 75 trial locations

Overseen By Grace Mausisa

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Alector Inc.

Must not be taking: Anticoagulants

Disqualifiers: Alzheimer's, Parkinson's, Cancer, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

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 people with frontotemporal dementia (FTD), a brain disorder affecting behavior and language. The goal is to determine if AL001 is safe and effective for individuals with a specific gene mutation linked to FTD. Participants will receive either the AL001 treatment or a placebo (a harmless pill with no active ingredients) every four weeks. This trial suits those with the progranulin gene mutation who are either showing symptoms of FTD or are at risk of developing them. Participants need someone to visit or care for them for at least five hours each week. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are currently using anticoagulant medications (blood thinners).

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that AL001 is generally safe and well-tolerated. Earlier studies found no major safety concerns. In these studies, individuals with a specific gene mutation linked to frontotemporal dementia took AL001, and no significant safety issues were reported. This suggests AL001 might be safe for humans, but ongoing research will provide more detailed safety information. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for frontotemporal dementia, which often focus on managing symptoms through behavioral therapies and medications like antidepressants or antipsychotics, AL001 offers a novel approach. AL001 is designed to target the root cause by modifying the levels of a specific protein called progranulin, which is linked to the progression of the disease. This targeted mechanism sets AL001 apart, potentially slowing or even halting the disease's progression rather than just alleviating symptoms. Researchers are excited because this could lead to more effective and sustainable treatment outcomes for patients.

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

Research has shown that AL001, also known as latozinemab, might help treat a type of dementia related to changes in the progranulin gene. AL001 increases the amount of progranulin, a protein that protects brain cells. Earlier studies found that AL001 was generally safe and well tolerated, with early signs of slowing brain damage. In this trial, participants will receive either AL001 or a placebo every four weeks. Recent study results highlighted AL001's potential benefits, although specific improvement rates were not detailed. Overall, the treatment seems promising in addressing the root cause of this type of dementia. ^{1 2 3 6 7}

Who Is on the Research Team?

TBD TBD

Principal Investigator

Are You a Good Fit for This Trial?

This trial is for people with a specific gene mutation linked to frontotemporal dementia (FTD), including those at risk of developing FTD or diagnosed with it. Participants need a study partner available for support, and they must not be pregnant, planning pregnancy, or have significant heart, liver, kidney diseases, uncontrolled diabetes or hypertension.

Inclusion Criteria

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).

I have a progranulin gene mutation and am at risk of or have been diagnosed with FTD.

Exclusion Criteria

You have had severe allergic reactions to certain types of medications made from human or animal proteins.

I have had cancer in the past 5 years.

I do not have uncontrolled high blood pressure, diabetes, thyroid, heart, liver, or kidney disease, and no significant brain disease other than FTD.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive AL001 or placebo intravenously every 4 weeks

96 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Assess the long-term safety and tolerability of AL001 in participants who have completed 96 weeks of treatment

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• AL001
• Placebo

Trial Overview

The trial tests the safety and effectiveness of AL001 in treating FTD compared to a placebo. Some participants will receive AL001 openly. It's designed as a phase 3 double-blind study where neither the researchers nor participants know who gets the real treatment versus placebo.

How Is the Trial Designed?

Group I: Open label - AL001 Experimental Treatment 1 Intervention

AL001 every 4 weeks

Group II: AL001 Experimental Treatment 1 Intervention

AL001 every 4 weeks

Group III: Placebo Placebo Group 1 Intervention

Placebo every 4 weeks

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

Published Research Related to This Trial

A review of 9 randomized controlled trials found that pharmacological treatments like SSRIs, trazodone, and stimulants may help reduce behavioral symptoms in frontotemporal dementias, but they do not improve cognitive function.

All medications studied were well tolerated by participants, indicating a favorable safety profile for these treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacological treatments for frontotemporal dementias: a systematic review of randomized controlled trials. Nardell, M., Tampi, RR.[2022]

In a 6-month study involving 16 patients with frontotemporal dementia, memantine at a dose of 20 mg/day was well-tolerated but showed limited efficacy, with most patients either unchanged or minimally worse in cognitive and behavioral assessments.

The study indicated a decline in cognitive performance as measured by the Alzheimer's Disease Assessment Scale, suggesting that while memantine is safe, its effectiveness in improving symptoms of frontotemporal dementia remains uncertain and warrants further investigation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A 6-month, open-label study of memantine in patients with frontotemporal dementia. Diehl-Schmid, J., Förstl, H., Perneczky, R., et al.[2013]

No effective pharmacological treatments have been proven to significantly alter the progression of frontotemporal dementia (FTD) spectrum disorders, highlighting a critical gap in current research.

A review of 23 randomized controlled trials revealed that many were small, short-term, and lacked precision in patient diagnosis, suggesting that future studies should incorporate genetic and molecular testing to better address the heterogeneity of FTD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Therapeutic trial design for frontotemporal dementia and related disorders. Desmarais, P., Rohrer, JD., Nguyen, QD., et al.[2021]

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.

mayo.edu

mayo.edu/research/clinical-trials/cls-20515235

A Study to Evaluate Effectiveness and Safety of AL001 in ...

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

7.

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 ...