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

Alector Inc.0 sites180 target enrollmentJune 30, 2020

Overview

No summary available.

Registry

who.int

Start Date

June 30, 2020

End Date

TBD

Last Updated

5 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

•Each participant must meet all of the following criteria to be enrolled in this study.
•Key Inclusion Criteria:
•Participant is a known carrier of a heterozygous loss\-of\-function GRN mutation causative of FTD, with either:
•A global CDR® plus NACC FTLD score of 0 and an elevated level of serum NfL as measured on the Simoa NF\-Light Advantage Kit assay (pre\-symptomatic participants) or,
•A global CDR® plus NACC FTLD score of 0\.5, 1, or 2; and 1 or more of the
•6 behavioral/cognitive symptoms required for a diagnosis of possible bvFTD, or a diagnosis of PPA (symptomatic participants).
•General Inclusion Criteria:
•2\. Presymptomatic participants (with a global CDR® plus NACC FTLD score of 0\) are 45 to 85 years of age, inclusive, at screening, or symptomatic participants (with a global CDR® plus NACC FTLD score of 0\.5,1, or 2\) are 25 to 85 years of age, inclusive, at screening
•3\. At screening, female participants must be nonpregnant and nonlactating, and 1 of the following conditions must apply:
•a. Participant is not a woman of childbearing potential (WOCBP) (either surgically sterilized or physiologically incapable of becoming pregnant, or at least 1\-year postmenopausal \[amenorrhea duration of 12 consecutive months with no identified cause other than menopause]).

•1\. Participant has 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.
•2\. Participant has a known mutation causative of neurodegenerative disorder(s) other than heterozygous loss\-of\-function GRN mutations causative of FTD.
•3\. Participant has a known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.
•4\. Participant has a history of moderate or severe substance use disorder within the past
•2 years, with the exception of nicotine, as defined by the Diagnostic and Statistical
•Manual of Mental Disorders, fifth edition criteria
•5\. Participant currently has or has had an acute illness that requires or required systemic antibiotics within 30 days prior to study treatment administration.
•6\. Participant has clinically significant vitamin B12 or folate deficiency (if treated, must be on a stable regimen for at least 3 months prior to study treatment administration).
•7\. Participant has untreated hypothyroidism (if treated, thyroid supplementation dose must be stable for at least 3 months with a normal thyroid\-stimulating hormone level prior to study treatment administration).
•8\. Participant has insufficiently controlled diabetes mellitus (e.g., hemoglobin A1C