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| Name | Class |
|---|---|
| Celerion | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AG10 single oral dose | Experimental | AG10 oral tablet, administered by mouth, once |
|
| Placebo single oral dose | Placebo Comparator | Placebo Oral Tablet, administered by mouth, once |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AG10 oral tablet | Drug | Active single ascending dose |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 30 days |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetic Assessments: T1/2 | Plasma half-life (t1/2) | 30 days |
| Pharmacokinetic Assessments: Tmax | Time to maximum concentration (Tmax) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Terry O'Reilly, M.D. | Celerion | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Celerion | Tempe | Arizona | 85283 | United States |
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| ID | Term |
|---|---|
| D028227 | Amyloid Neuropathies, Familial |
| ID | Term |
|---|---|
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D017772 | Amyloid Neuropathies |
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Subjects will be randomized with an overall ratio of 3:1 to AG10: placebo within each cohort
| Placebo Oral Tablet | Drug | Placebo single dose |
|
|
| 30 days |
| Pharmacokinetic Assessments: Cmax | Maximum concentration (Cmax) | 30 days |
| Pharmacokinetic Assessments: Cmin | Cmin | 30 days |
| Pharmacokinetic Assessments: AUC | Area under the plasma concentration-time curve (AUC) | 30 days |
| Pharmacokinetic Assessments: Clearance | Apparent clearance (CL/F) | 30 days |
| Pharmacokinetic Assessments: volume of distribution | Apparent volume of distribution (Vss/F) | 30 days |
| Pharmacodynamic Assessments: Assessments of TTR stabilization will be listed and summarized by part, treatment, and time point using appropriate descriptive statistics. | 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). | 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) | 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). | 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. | 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. | 30 days |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028226 | Amyloidosis, Familial |
| D008661 | Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000686 | Amyloidosis |
| D057165 | Proteostasis Deficiencies |