Clinical Trial: Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA M
Brief Summary: Study to compare the efficacy and safety of AT1001 and enzyme replacement therapy (ERT) in male and female patients with Fabry disease who are currently receiving ERT and who have AT1001-responsive GLA mutations.
Detailed Summary:
Sponsor: Amicus Therapeutics
Current Primary Outcome: renal function assessed by iohexol Glomerular Filtration Rate (GFR) [ Time Frame: 18 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- renal function (assessed by estimated GFR and 24-hour urine protein) [ Time Frame: 18 months ]
- composite clinical outcome (assessed by time to occurrence of renal, cardiac, cerebrovascular events or death) [ Time Frame: 18 months ]
- cardiac function (assessed by echocardiography) [ Time Frame: 18 months ]
- patient reported outcomes (pain and quality of life) [ Time Frame: 18 months ]
Original Secondary Outcome: Same as current
Information By: Amicus Therapeutics
Dates:
Date Received: October 6, 2010
Date Started: December 2010
Date Completion:
Last Updated: June 25, 2015
Last Verified: June 2015
