Albuterol in Individuals With Late Onset Pompe Disease (LOPD)
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.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|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 Replacement Therapy|
- Number of Participants with Adverse Events [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]
- Change in forced vital capacity at 3 months [ Time Frame: Baseline and week 12 ] [ Designated as safety issue: No ]
- Change in 6 minute walk test in 6 months. [ Time Frame: Baseline and 24 weeks ] [ Designated as safety issue: No ]Assessed by physical therapist.
|Study Start Date:||June 2012|
|Estimated Study Completion Date:||December 2013|
|Primary Completion Date:||April 2013 (Final data collection date for primary outcome measure)|
This study includes the off-label administration of oral albuterol (4 mg pill) and tracking the effect of motor function at two additional visits, 6 and 12 weeks following the initiation of the study drug. The initial dose of albuterol will be a 4 mg daily for the first 6 weeks. If the 4 mg is well tolerated, the dose will be increased to 8 mg at the 6 week visit.
Albuterol 4 mg
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27710|
|Principal Investigator:||Dwight D Koeberl, MD, PhD||Duke University|