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

Type 1 Gaucher Disease Clinical Trial

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

Status Terminated

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Gaucher Disease
• Type 1 Gaucher Disease

• NCT number: NCT00875160
• Study type: Interventional
• Source: Amicus Therapeutics
• Status: Terminated
• Phase: Phase 1
• Start date: April 2009
• Completion date: July 2009