NCT00875160

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

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

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Type 1 Gaucher Disease

Interventions

Type	Name	Description
DRUG	AT2101	225mg (nine 25mg capsules total dose) to be taken by mouth every day for 9 days (Study Days 1, 3-10)

Timeline

• Start date: 2009-04-01
• Primary completion: 2009-07-01
• Completion: 2009-07-01
• First posted: 2009-04-03
• Last updated: 2010-08-19

Locations

2 sites across 2 countries: United States, United Kingdom