A Study of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)

  • Principal Inviestigator: Peter Ljubenkov, MD
  • Sponsor: Alector Inc.
  • Status: Recruiting
  • Official Study Title: 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
  • ClinicalTrials.gov Identifier: NCT03987295
  • Conditions Studied: Frontotemporal dementia (FTD)
  • Intervention: AL001
  • Phase: Phase 2
  • Duration of Participation:

Purpose of the Study

A Phase 2 open-label study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia (FTD).


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, physical examinations (PEs), laboratory tests, ECGs, and vital signs
  • Participant is a carrier of a loss of function progranulin gene (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

What to Expect

Testing: Neurological and physical examinations, cognitive testing and neuropsychiatric assessments, telephone calls, blood and urine specimen collection, and vital signs

The Frequency of Visits: Every 4 weeks for up to 96 weeks

Materials Needed Before Evaluation: Genetic testing for a loss of function progranulin gene (GRN) mutation or carrier of a hexanucleotide repeat expansion C9orf72 mutation

Costs: No costs will be charged for any of the study procedures. Parking will be validated at UCSF public garages for all study visits.

Contact Information

Coordinator: Irina Gorodetskaya[email protected], 415.476.2941
Clinical Trials Nurse: Mary Koestler, RN, PhD, CCRC[email protected], 415.476.0661