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AL001 for Frontotemporal Dementia


This Phase II study will evaluate the safety and tolerability of AL001, a drug being tested in participants with frontotemporal dementia caused by a granulin genetic mutation (FTD-GRN) or a C9orf72 genetic mutation. AL001, a monoclonal antibody, increases the level of progranulin, a protein whose production is lowered in FTD-GRN. About 5-10 percent of people with FTD have a mutation in the progranulin gene; another 5-10 percent have a mutation in the C9orf72 gene.

Minimum Age Maximum Age Gender Healthy Volunteers
18 Years 80 Years All No
September 27, 2019
May 30, 2023

  • Female participants must be nonpregnant and nonlactating
  • Good physical health; no clinically significant findings from medical history, physical exam, laboratory tests, electrocardiogram, and vital signs
  • Carrier of a loss of function GRN mutation or carrier of a hexanucleotide repeat expansion C9orf72 mutation

  • Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins
  • History of alcohol abuse or substance abuse
  • Living in a skilled nursing facility, convalescent home, or long-term care facility

Participants will take the study drug, administered intravenously, every 4 weeks for up to 48 weeks. Researchers will measure the concentration of AL001 in the blood at specified time points, as well as the drug's effects on biomarkers of neurodegeneration and on cognition.

Name City State Zip Status Primary Contact
University of Alabama
Birmingham Alabama 35294 Withdrawn

San Francisco California 94158 Recruiting Study Coordinator
Mayo Clinic
Rochester Minnesota 55905 Active, not recruiting

University of Pennsylvania
Philadelphia Pennsylvania 19104 Active, not recruiting

Lawson Health Research Institute, St. Joseph's
London Ontario N6A 4V2 Recruiting Study Coordinator
Sunnybrook Health Sciences Centre
Toronto Ontario M4N 3M5 Recruiting Study Coordinator

Alector Inc.

Name Role Affiliation
Peter Ljubenkov, MD Principal Investigator University of California, San Francisco

Name Phone Email
Study Lead 415-231-5660


A Phase 2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in Heterozygous Carriers of Granulin or C9ORF72 Mutations Causative of Frontotemporal Dementia