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

Duke University

Status

Completed

Conditions

Glycogen Storage Disease Type II

Treatments

Device: RMT therapy using modified RMT device

Device: Sham-RMT therapy using modified RMT device

Study type

Interventional

Funder types

Other

NIH

Identifiers

NCT02801539

Pro00068728

1R21AR069880-01 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

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.

Enrollment

22 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

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