Clinical Trial: Study to Evaluate the Safety of AT2220 in Pompe Disease

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

Official Title: An Open-Label, Multicenter, Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Three Dosing Regimens of Oral AT2220 in Patients With Pompe Dise

Brief Summary: The main purpose of this study is to determine the safety and tolerability of three different doses of AT2220 in people affected by Pompe disease. The study will also evaluate the effects of AT2220 on functional parameters in Pompe disease.

Detailed Summary: Subjects meeting all eligibility criteria will undergo physical examination, electrocardiogram (ECG), spirometry, muscular strength test, functional muscle test, 6-minute walk test (6MWT) (when appropriate), laboratory tests, MRI and muscle (needle) biopsy. Quality of life will be assessed via SF-36 questionnaire. Functional ability and level of handicap will be assessed by Rotterdam handicap scale.
Sponsor: Amicus Therapeutics

Current Primary Outcome: Treatment-emergent Adverse Events [ Time Frame: 11 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Change in functional parameters from Baseline to End of Study [ Time Frame: 11 weeks ]

Original Secondary Outcome: Same as current

Information By: Amicus Therapeutics

Dates:
Date Received: May 30, 2008
Date Started: November 2008
Date Completion:
Last Updated: May 16, 2011
Last Verified: May 2011