Summary

Eligibility
for people ages 18-85 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion
Principal Investigator
Peter Ljubenkov, MD

Description

Summary

A phase 3 double blind, placebo controlled study evaluating the efficacy and safety of AL001 in participants at risk for or with frontotemporal dementia due to heterozygous mutations in the progranulin gene.

Official Title

A Phase 3, Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene

Details

This is a phase 3 double blind, placebo controlled study evaluating the efficacy and safety of AL001 administered intravenously in participants at risk for or with frontotemporal dementia due to heterozygous mutations in the progranulin gene.

Keywords

Frontotemporal Dementia Dementia Aphasia, Primary Progressive Pick Disease of the Brain

Eligibility

You can join if…

Open to people ages 18-85

  • Persons with a progranulin gene mutation and at risk of developing FTD symptoms as evidenced by a biomarker, or persons with a progranulin gene mutation and diagnosed with FTD.
  • If symptomatic, one of the criteria for the diagnosis of probable behavioral variant FTD, or FTD-semantic subtype or FTD-Progressive nonfluent aphasia.
  • Study partner who consents to study participation and who cares for/visits the participant daily for at least 5 hours per week.
  • Written informed consent must be obtained and documented (from the participant or, where jurisdictions allow it, from their legal decision maker).

You CAN'T join if...

  • Dementia due to a condition other than FTD including, but not limited to, Alzheimer's disease, Parkinson's disease, dementia with Lewy bodies, Huntington disease, or vascular dementia.
  • Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.
  • Current uncontrolled hypertension, diabetes mellitus or thyroid disease. Clinically significant heart disease, liver disease or kidney disease. History or evidence of clinically significant brain disease other than FTD.
  • Females who are pregnant or breastfeeding, or planning to conceive within the study period.
  • Any experimental vaccine or gene therapy.
  • History of unresolved cancer.
  • Current use of anticoagulant medications (e.g., coumadin, heparinoids, apixaban).
  • Residence in a skilled nursing facility, convalescent home, or long term care facility at screening; or requires continuous nursing care.

Locations

  • University of California San Francisco not yet accepting patients
    San Francisco California 94143 United States
  • University of California San Diego accepting new patients
    La Jolla California 92093-0648 United States

Lead Scientist at UCSF

  • Peter Ljubenkov, MD
    Assistant Professor, Neurology. Authored (or co-authored) 17 research publications.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Alector Inc.
ID
NCT04374136
Phase
Phase 3
Study Type
Interventional
Last Updated