NCT01859624

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Duke University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Pompe Disease

Interventions

Type	Name	Description
DRUG	Albuterol	Albuterol 4 mg

Timeline

• Start date: 2012-06-01
• Primary completion: 2013-04-01
• Completion: 2013-04-01
• First posted: 2013-05-22
• Last updated: 2019-07-05

Locations

1 site across 1 country: United States