A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
This is an open-label study designed to assess if AT2101 is safe in patients with Gaucher disease and how AT2101 gets through the body after it is taken by mouth. The study is being offered to adult patients with type 1 Gaucher disease who are currently receiving a stable dose of enzyme replacement therapy (ERT) with imiglucerase. During the study, subjects will not be receiving ERT (up to 35 days).
The study consists of a screening period (~14 days), a treatment period (12 days) and a follow-up period (7 days after last dose). At two points in the study, subjects will be housed in an in-patient treatment facility for 3 days/2 nights to accommodate all necessary blood draws.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||An Open-Label Study to Assess the Pharmacokinetics, Safety, and Pharmacodynamics of Repeated Doses of Orally Administered AT2101 in Adult Patients With Type 1 Gaucher Disease|
- To evaluate single-dose and multiple-dose pharmacokinetics of isofagomine following the oral administration of AT2101 in patients with type 1 Gaucher disease [ Time Frame: Multiple times for up to three days following Day 1 and Day 10 ] [ Designated as safety issue: No ]
- To evaluate the safety and tolerability of oral AT2101 administered in patients with type 1 Gaucher disease [ Time Frame: Daily ] [ Designated as safety issue: Yes ]
- To evaluate the pharmacodynamic effect of WBC GCase of oral AT2101 administered in patients with type 1 Gaucher disease. [ Time Frame: Days 1, 5 & 7 (optional), 10 and 17 ] [ Designated as safety issue: No ]
|Study Start Date:||April 2009|
|Study Completion Date:||July 2009|
|Primary Completion Date:||July 2009 (Final data collection date for primary outcome measure)|
225mg (nine 25mg capsules total dose) to be taken by mouth every day for 9 days (Study Days 1, 3-10)
While on the study, subjects will be required to refrain from consuming the following foods and beverages:
- Caffeine: 24 hours before admission and throughout the in-patient stay;
- Alcohol: 48 hours before admission and throughout the in-patient stay. A maximum of 2 units/day will be allowed during the out-patient part of the study (1 unit of alcohol = wind (125mL)=spirits(25mL)=beer (284mL);
- Vitamins: throughout the in-patient periods.
Subjects will also be restricted to taking medications or herbal products during the study except if the Principal Investigator permits.
Subjects will not engage in strenuous activity at any time during the in-patient periods.
|United States, Florida|
|Comprehensive Phase One|
|Miramar, Florida, United States, 33025|
|Guy's Drug Research Unit|
|London, United Kingdom, SE1 1YR|
|Study Director:||Eugene Schneider, MD||Amicus Therapeutics|