A Study to Evaluate Effectiveness and Safety of AL001 in Frontotemporal Dementia

Overview

About this study

The primary purpose of this study is to evaluate the efficacy of AL001 compared with placebo in carriers of progranulin gene (GRN) mutations causative of frontotemporal dementia (FTD) as measured by the Clinical Dementia Rating Dementia Staging Instrument PLUS National Alzheimer’s Disease Coordinating Center frontotemporal lobar degeneration Behavior & Language Domains Sum of Boxes (CDR® plus NACC FTLD-SB).

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:

- 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 or more of the criteria for the diagnosis of possible behavioral
variant FTD, or a diagnosis of Primary Progressive 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).

Exclusion Criteria:

- 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 cancer within the last 5 years.

- 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.

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 4/2/2024. Questions regarding updates should be directed to the study team contact.

 

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.

Closed for enrollment

Contact information:

Kevin Nelson

(507) 284-9295

Nelson.Kevin1@mayo.edu

More information

Publications

Publications are currently not available
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CLS-20515235

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