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

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

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

Brief 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 Summary:

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.


Sponsor: Amicus Therapeutics

Current Primary Outcome: 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 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • To evaluate the safety and tolerability of oral AT2101 administered in patients with type 1 Gaucher disease [ Time Frame: Daily ]
  • 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 ]


Original Secondary Outcome: Same as current

Information By: Amicus Therapeutics

Dates:
Date Received: April 2, 2009
Date Started: April 2009
Date Completion:
Last Updated: August 17, 2010
Last Verified: August 2010