AL001 for Frontotemporal Dementia

Recruiting at 68 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

110 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing AL001, a drug given through an IV, to see if it can help people with a genetic mutation that causes frontotemporal dementia. The goal is to find out if AL001 can improve brain health in these individuals.

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).

What safety data exists for AL001 or similar treatments for frontotemporal dementia?

The available data from trials on similar treatments like SSRIs, trazodone, and amphetamines indicate they were generally well tolerated, with some reports of side effects like fatigue, dizziness, and low blood pressure. ^{1 2 3 4 5}

Research Team

TBD TBD

Principal Investigator

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Pharmacological treatments for frontotemporal dementias: a systematic review of randomized controlled trials. [2022]

2. Germany pubmed.ncbi.nlm.nih.gov

[Frontotemporal dementia: neurotransmitter and clinical symptoms with focus on therapeutic targets]. [2009]

3. England pubmed.ncbi.nlm.nih.gov

A 6-month, open-label study of memantine in patients with frontotemporal dementia. [2013]

4. England pubmed.ncbi.nlm.nih.gov

Therapeutic trial design for frontotemporal dementia and related disorders. [2021]

5. Switzerland pubmed.ncbi.nlm.nih.gov

Efficacy of fluvoxamine as a treatment for behavioral symptoms in frontotemporal lobar degeneration patients. [2022]