Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01730469 | Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function | PHASE1 | COMPLETED | 32 | — | — | Aug 1, 2011 | Apr 1, 2012 | Aug 3, 2017 | 4 | United States |
| NCT01730482 | A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014) | PHASE1 | COMPLETED | 6 | — | — | Aug 1, 2011 | Sep 1, 2011 | Dec 18, 2013 | - | — |
Adverse events will be evaluated from Day 1 to the end of study (Day 10 +1).
Clinical laboratory evaluations (hematology, clinical chemistry, urinalysis, Hepatitis A and HIV screen) will be evaluated from screening to the end of the study.
Vital signs (oral temperature, respiratory rate, and seated blood pressure) will be performed from screening to the end of the study.
Physical examination (general appearance, skin, thorax/lungs, cardiovascular and abdomen) will be performed from screening to the end of the study.
Electrocardiogram (ECG) measures the electrical activity of the heart and the hearts' rhythm. All subjects will undergo ECG testing.
Urine will be collected: pre-dose (-12-0 hours), 0-12 and 12-24 hours on Day 1, 24-hourly on Days 2-10; the last sample will be collected on Day 11. Total radioactivity excreted in urine will be calculated for the entire collection period. The excretion rate will be calculated from total radioactivity collected in urine, divided by the duration of collection. The percentage of dose excreted in urine will be calculated from the total amount excreted in urine divided by the dose administered, multiplied by 100.
Feces will be collected: predose (-24 to 0 hours); all bowel movements post-dose will be collected on Days 1 to 10; the last sample will be collected on Day 11. Total radioactivity excreted in feces will be calculated for the entire collection period. The excretion rate will be calculated from total radioactivity collected in feces, divided by the duration of collection. The percentage of dose excreted in feces will be calculated from the total amount excreted in feces divided by the dose administered, multiplied by 100.
Expired air will be collected: pre-dose and at 2, 4, 6 and 24 hours after dosing. Each subject will blow into a measured volume of sodium hydroxide solution containing phenolphthalein, until an indicator changes color, showing that the sodium hydroxide is saturated. This sodium hydroxide solution will be analysed for radioactive content. If radioactivity is detected the total amount in expired air during the collection period will be calculated.
Blood samples will be collected: Pre-dose, at 30 minutes, and 1, 1.5, 2, 3, 4, 6, 8, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216 and 240 hours after dosing. Non-compartmental pharmacokinetic parameters will be calculated for plasma AT1001: area under the drug concentration-time curve (AUC) from time zero to the time of the last measurable concentration, AUC from time zero to infinity, maximum observed drug concentration, time of the maximum drug concentration, apparent terminal elimination rate constant and apparent elimination half life.
Blood samples will be collected: Pre-dose, at 30 minutes, and 1, 1.5, 2, 3, 4, 6, 8, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 192, 216 and 240 hours after dosing. Non-compartmental pharmacokinetic parameters will be calculated for plasma radioactivity: area under the drug concentration-time curve (AUC) from time zero to the time of the last measurable concentration, AUC from time zero to infinity, maximum observed drug concentration, time of the maximum drug concentration, apparent terminal elimination rate constant and apparent elimination half life.
| Arm | Type | Description |
|---|---|---|
| AT1001 150 mg | EXPERIMENTAL | Each subject will receive a single oral dose of AT1001 150 mg administered orally with 240 mL room temperature water after at least a 4-hour fast |
| [14C] AT1001 Arm | EXPERIMENTAL | Each subject will receive a single oral dose of 150 mg of \[14C\] AT1001 as an aqueous solution containing 1 μCi AT1001 on Study Day 1. |
| Name | Type | Description |
|---|---|---|
| AT1001 150 mg | DRUG | AT1001 150mg is available as a capsule |
| [14C] AT1001 | DRUG | 150 mg of \[14C\] labelled AT1001 will be administered as a solution (prepared by weighing 150 mg of AT1001 powder and dissolving in 100 mL of water). The entire solution will be swallowed orally. After ingestion of study medication, the dosing bottle will be rinsed with 50 mL of water and this will be ingested by the subject. This rinse procedure should be performed twice. Following the second rinse ingestion, the subjects should be instructed to drink additional water to bring the total volume ingested to 240 mL. |
Inclusion Criteria All subjects * males or females aged 18 to 70 years inclusive (subjects with normal renal function, mild or moderate renal impairment), and 18 to 75 years inclusive (subjects with severe renal impairment) * body mass index 18.0 to 40.0 kilogram (kg)/square meter (m\^2) inclusive ...