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

Overview

About this study

The puppse pf tjos stidu os tp evaluate the safety, tolerability, pharmacokinets (PK) and pharmacodynamics (PD) of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of 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 Contact

Rochester, Minn.

Mayo Clinic principal investigator

Bradley Boeve, M.D.

Open for enrollment

Contact information:

Neurology Study Team

(507) 284-1324

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available