Clinical Trial: Albuterol in Individuals With Late Onset Pompe Disease (LOPD)
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Clinical Investigation of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease, Whether or Not Receiving Enzyme Replacemen
Brief Summary: Albuterol is a drug approved by the US Food and Drug Administration (FDA) for treating breathing problems such as asthma. Studies have shown that albuterol may be beneficial in improving muscle function in people with late-onset Pompe disease. The purpose of this study is to evaluate whether albuterol is safe and effective for improving muscle function in people with late-onset Pompe disease, whether or not they are receiving enzyme replacement therapy (ERT). For this study, albuterol is considered an investigational drug. The word "investigational" means albuterol is not approved by the FDA for individuals with late-onset Pompe disease.
Detailed Summary:
Sponsor: Duke University
Current Primary Outcome: Number of Participants with Adverse Events [ Time Frame: 24 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Change in forced vital capacity at 3 months [ Time Frame: Baseline and week 12 ]
- Change in 6 minute walk test in 6 months. [ Time Frame: Baseline and 24 weeks ]Assessed by physical therapist.
Original Secondary Outcome: Same as current
Information By: Duke University
Dates:
Date Received: May 8, 2013
Date Started: June 2012
Date Completion:
Last Updated: February 11, 2016
Last Verified: January 2016
