Principal Investigator
Enrollment Status
Clinical Trial ID
Clinical Trial 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.
Phase
Phase 3
Funding Agency/Sponsor
Industry
Disease
NeuroPsychology
Enrollment Eligibility
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.