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

Completed · Not applicable · Has a placebo group

Conditions studied: Glycogen Storage Disease Type II

In brief

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.

Key facts

Study ID

NCT02801539

Run by

Duke University

People needed

22

Starts

2017-02-02

Expected to finish

2019-04-17

Last updated by the study team

2019-07-30

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age ≥ 18 years
• Diagnosis of LOPD
• On enzyme replacement therapy for ≥ 26 weeks at pretest
• Able to follow directions for study participation
• Able to complete a home-based RMT regimen

You may not qualify if…

• Neurodegenerative conditions (e.g. stroke, dementia) or other serious neurologic condition that would prevent meaningful study participation as determined at the discretion of the principle investigator
• Inability to give legally effective consent
• Inability to read and understand English

Where it is running

• Duke University Medical Center — Durham, North Carolina, United States

Full record on ClinicalTrials.gov

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