Clinical Trial: A Long-Term Extension Study of AT2101 in Type 1 Gaucher Patients

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open-label, Multicenter, Long-term Extension Study to Assess the Safety, Efficacy, and Pharmacodynamics of AT2101 in Adult Patients With Type 1 Gaucher Disease

Brief Summary: This study will measure the long-term safety of AT2101 and its effects on hemoglobin, platelets, liver and spleen volume, and bone density. This study will also look at the effects of AT2101 on beta-glucocerebrosidase levels, glucosylceramide levels, and other blood markers of Gaucher disease.

Detailed Summary:

This is an open-label, long-term extension study designed to assess the long-term safety, efficacy and pharmacodynamics of AT2101 in patients with type 1 Gaucher disease who successfully complete Study GAU-CL-202. Participants may enter this study immediately upon completion of Study GAU-CL-202, or at any later time point. Each participant will continue to receive AT2101 at the same dose level and regimen as in the previous study.

Study visits will occur every 3 months. At every visit, safety evaluations will be performed and blood samples will be collected for pharmacodynamic tests. At the completion of every year of treatment in this study, or at the early termination visit, participants will undergo a comprehensive physical examination, MRIs of liver, spleen and femoral bones, DEXA scans of femoral bones and lumbar spine, and complete the SF-36 questionnaire. Duration of treatment will depend on the time of each participant's enrollment in the study. Participants will be contacted approximately 1 month after study completion for assessment of adverse events.


Sponsor: Amicus Therapeutics

Current Primary Outcome: The primary objective of the study is to evaluate the long-term safety of orally administered AT2101 in adult patients with type 1 Gaucher disease. [ Time Frame: Every 3 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • The secondary objective of the study is to assess the long-term efficacy of orally administered AT2101 in adult patients with type 1 Gaucher disease [ Time Frame: Every 3 months ]
  • The tertiary objective of the study is to assess the pharmacodynamics of orally administered AT2101 in adult patients with type 1 Gaucher disease [ Time Frame: Every 3 months ]


Original Secondary Outcome: Same as current

Information By: Amicus Therapeutics

Dates:
Date Received: December 22, 2008
Date Started: January 2009
Date Completion:
Last Updated: September 7, 2012
Last Verified: September 2012