A Study to Evaluate Safety of Long-term AL001 Dosing in FTD Patients


About this study

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of AL001 in participants with a  frontotemporal dementia.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria: 

  • At screening, female participants must be nonpregnant and nonlactating.
  • In good physical health on the basis of no clinically significant findings from medical history, PEs, laboratory tests, ECGs, and vital signs.
  • Participant is a carrier of a loss of function GRN mutation or carrier of a hexanucleotide repeat expansion C9orf72 mutation.

Exclusion Criteria:

  • 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.
  • Participant resides in a skilled nursing facility, convalescent home, or long term care facility.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Bradley Boeve, M.D.

Closed for enrollment

More information


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