This is a single center, prospective, randomized, placebo-controlled study of AG10 in healthy adult subjects
Up to 48 healthy volunteers will be given a single dose of AG10 or placebo and be monitored for safety and tolerability over a 5-day period. Up to 48 healthy volunteers will be given multiple doses of AG10 or placebo and be monitored for safety and tolerability over a 15-day period.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
56
Active single ascending dose
Placebo single dose
Celerion
Tempe, Arizona, United States
Safety & tolerability: individual and summary blood pressures, heart rate, ECG and lab data presented in tabular form with descriptive statistics. Adverse events will be tabulated and summarized by Part A (SAD) vs. B (MAD), and treatment.
To evaluate the safety and tolerability of single and multiple doses of AG10 administered to healthy adult subjects
Time frame: 30 days
Pharmacokinetic Assessments: T1/2
Plasma half-life (t1/2)
Time frame: 30 days
Pharmacokinetic Assessments: Tmax
Time to maximum concentration (Tmax)
Time frame: 30 days
Pharmacokinetic Assessments: Cmax
Maximum concentration (Cmax)
Time frame: 30 days
Pharmacokinetic Assessments: Cmin
Cmin
Time frame: 30 days
Pharmacokinetic Assessments: AUC
Area under the plasma concentration-time curve (AUC)
Time frame: 30 days
Pharmacokinetic Assessments: Clearance
Apparent clearance (CL/F)
Time frame: 30 days
Pharmacokinetic Assessments: volume of distribution
Apparent volume of distribution (Vss/F)
Time frame: 30 days
Pharmacodynamic Assessments: Assessments of TTR stabilization will be listed and summarized by part, treatment, and time point using appropriate descriptive statistics.
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AG10 binding to and/or stabilization of TTR will be evaluated by established ex vivo assays, including Fluorescent Polarization Exclusion Assay (FPE) and Immunoblotting (Western Blot) and quantitation of prealbumin (TTR).
Time frame: 30 days
Pharmacodynamic Assessments: Western blot
AG10 binding to and/or stabilization of TTR will be evaluated by established ex vivo assays: Immunoblotting (Western Blot)
Time frame: 30 days
Pharmacodynamic Assessments: prealbumin
AG10 binding to and/or stabilization of TTR will be evaluated by established ex vivo assays: quantitation of prealbumin (TTR).
Time frame: 30 days
Food effect: AUC
To evaluate the effect of food on the PK of AG10. The log transformed values of total AUC will be analyzed using a linear mixed effect model with formulation, period, sequence, and carryover as fixed effects and subject as a random effect.
Time frame: 30 days
Food effect: Cmax
To evaluate the effect of food on the PK of AG10. The log transformed values of Cmax will be analyzed using a linear mixed effect model with formulation, period, sequence, and carryover as fixed effects and subject as a random effect.
Time frame: 30 days