A first in human phase 1 study in healthy volunteers and participants with Granulin (GRN) mutation causative of frontotemporal dementia (FTD) to assess AL001 safety, tolerability, pharmacokinetics, and pharmacodynamics
This is a first-in-human Phase 1 study designed to investigate the safety, tolerability, PK and PD of intravenously administered AL001 in Healthy Volunteers and FTD-GRN mutation carriers.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
64
University of Alabama
Birmingham, Alabama, United States
UCSF
San Francisco, California, United States
Study site
Orlando, Florida, United States
Mayo Clinic
Rochester, Minnesota, United States
Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs)
Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods.
Time frame: 85 days
Pharmacokinetics (PK) of AL001
Serum and cerebrospinal fluid (CSF) concentration of AL001 at specified time points
Time frame: 85 days
Maximum plasma concentration (Cmax) for AL001
Evaluate Cmax for serum and CSF concentration of AL001 at specified time points
Time frame: 85 days
Area under the curve concentration (AUC) for AL001
Evaluate AUC for serum and CSF concentration of AL001 at specified time points
Time frame: 85 days
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University of Pennsylvania
Philadelphia, Pennsylvania, United States
Lawson Health Research Institute, St. Joseph's
London, Ontario, Canada
Sunnybrook Health Sciences Centre
Toronto, Canada
University College London
London, United Kingdom