AL001 for Frontotemporal Dementia

Recruiting at 11 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

33 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Peter Ljubenkov, MD

Principal Investigator

University of California, San Francisco

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

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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