Clinical Trial: Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase 1/2 Double-Blind Study of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy

Brief Summary: In this study the study team proposes to investigate the efficacy of albuterol on motor function of individuals with Late Onset Pompe Disease (LOPD) who are receiving enzyme replacement therapy, given albuterol was well-tolerated in patients with Late Onset Pompe Disease.

Detailed Summary:
Sponsor: Duke University

Current Primary Outcome: Number of participants with Adverse Events. [ Time Frame: 52 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Change in forced vital capacity from pulmonary function tests at 30 weeks and 52 weeks. [ Time Frame: Baseline and weeks 30 and 52. ]
• Change in 6 minute walk test [ Time Frame: Weeks 6 and 52. ]
  Assessed by physical therapist

Original Secondary Outcome: Same as current

Information By: Duke University

Dates:
Date Received: June 21, 2013
Date Started: June 2013
Date Completion:
Last Updated: January 4, 2017
Last Verified: October 2016