The purposes of this study are to investigate the safety, tolerability, and pharmacodynamics of FRM-0334 in subjects with prodromal to moderate frontotemporal dementia with granulin mutation.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
30
UCSF Memory and Aging Center
San Francisco, California, United States
Compass Research, LLC
Orlando, Florida, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Evaluate the safety and tolerability of FRM-0334
* Number and percentage of subjects with AEs * Number and percentage of subjects with SAEs * Number and percentage of subjects who discontinue due to AEs * Number and percentage of subject deaths
Time frame: Baseline to Day 28 or Early Termination
Assess the pharmacodynamic (PD) effects of FRM-0334 on the change from baseline in plasma concentrations of progranulin (PGRN) after 28 days
Time frame: Baseline to Day 28 or Early Termination
Assess the pharmacodynamic effects of FRM-0334 on the change from baseline in cerebrospinal fluid (CSF) concentrations of PGRN after 28 days
Change in CSF progranulin concentration from baseline
Time frame: Baseline and Day 28
Characterize the plasma concentrations of FRM-0334 and metabolites following once daily dosing for 28 days
Cmax, Ctrough, tmax, t1/2, λz, AUC(0-τ), CL/F, Cav in plasma
Time frame: Day 1 to Day 28 or Early Termination
Characterize the CSF concentrations of FRM-0334 and metabolites following once daily dosing for 28 days
Cmax, Ctrough, tmax, t1/2, AUC0-τ, CL/F, Cav in CSF
Time frame: Day 1 to Day 28 or Early Termination
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