A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)
Frontotemporal Dementia / Posted 2 years ago
A Phase 2 open label study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia.
Inclusion Criteria :
Ages Eligible for Study: 18 Years to 85 Years (Adult, Older Adult)
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
- 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
- Study end date : 30/05/2023
- Principal Investigator : Peter Ljubenkov, MD University of California