AL001 for Frontotemporal Dementia
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.
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.12345
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.12467
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive intravenous administration of AL001 every 4 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension (optional)
Participants may opt into continuation of treatment long-term to assess safety and tolerability
What Are the Treatments Tested in This Trial?
Interventions
- AL001
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alector Inc.
Lead Sponsor
GlaxoSmithKline
Industry Sponsor
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