- Principal Inviestigator: Peter Ljubenkov, MD
- Sponsor: Alector Inc.
- Status: Recruiting
- Official Study Title: A Phase 2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in Heterozygous Carriers of Granulin or C9orf72 Mutations Causative of Frontotemporal Dementia
- ClinicalTrials.gov Identifier: NCT03987295
- Conditions Studied: Frontotemporal dementia (FTD)
- Intervention: AL001
- Phase: Phase 2
- Duration of Participation:
Purpose of the Study
A Phase 2 open-label study evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia (FTD).
Eligibility
Inclusion Criteria:
- 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
What to Expect
Testing: Neurological and physical examinations, cognitive testing and neuropsychiatric assessments, telephone calls, blood and urine specimen collection, and vital signs
The Frequency of Visits: Every 4 weeks for up to 96 weeks
Materials Needed Before Evaluation: Genetic testing for a loss of function progranulin gene (GRN) mutation or carrier of a hexanucleotide repeat expansion C9orf72 mutation
Costs: No costs will be charged for any of the study procedures. Parking will be validated at UCSF public garages for all study visits.
Contact Information
Coordinator: [email protected]
Clinical Trials Nurse: Whitney Walker, MS, CNS, RN – [email protected], 415.353.7532
