Clinical Trial: Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Respiratory Muscle Training in Late-Onset Pompe Disease

Brief Summary: This study is being done to test the effects of respiratory muscle training (RMT) in patients with late-onset Pompe Disease (LOPD) who have weakness of their breathing muscles. The results of this study will help design future research studies about RMT in LOPD. The goals of this study are to decide if sham-RMT is a useful control condition for RMT and to choose the best ways to measure the health benefits of RMT in LOPD.

Detailed Summary:
Sponsor: Duke University

Current Primary Outcome:

• Change in respiratory strength [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  RMT to sham-RMT comparison of maximum inspiratory pressures (MIP). Change pretest to posttest is primary measure (baseline to 12 weeks).
• Feasibility of sham-RMT, as measured by program adherence [ Time Frame: 12 weeks ]
  As measured by program adherence (good is considered greater than or equal to 80%). Helps determines if a future efficacy trial is warranted and if sham-RMT may be a good control condition for a future trial.

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Change in maximum expiratory pressures (MEP) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  Change pretest to posttest
• Change in 6-minute walk test (6MWT) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  Change pretest to posttest
• Change in Gait, Stairs, Gower, and Chair (GSGC) scale [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  Change pretest to posttest
• Change in peak cough flow (PCF) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  Change pretest to posttest
• Change in Rasch-built Pompe-specific Activity Score (R-PAct) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  Change pretest to posttest
• Change in diaphragm thickness [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  change pretest to posttest

Original Secondary Outcome:

• Change in maximum expiratory pressures (MEP) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  Change pretest to posttest
• Change in 6-minute walk test (6MWT) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  Change pretest to posttest
• Change in Gait, Stairs, Gower, and Chair (GSGC) scale [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  Change pretest to posttest
• Change in peak cough flow (PCF) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  Change pretest to posttest
• Change in Rasch-built Pompe-specific Activity Score (R-PAct) [ Time Frame: baseline, 12 weeks, 6 months, 9 months ]
  Change pretest to posttest

Information By: Duke University

Dates:
Date Received: June 13, 2016
Date Started: February 2, 2017
Date Completion: June 2019
Last Updated: May 18, 2017
Last Verified: March 2017