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

Not Applicable

Completed

• Conditions: Glycogen Storage Disease Type II
• Interventions: Device: Sham-RMT therapy using modified RMT device; Device: RMT therapy using modified RMT device

• Registration Number: NCT02801539
• Lead Sponsor: Duke University

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 Description

Not available

Study Timeline

• Study First Submitted: 2016-06-13
• Study First Submitted QC: 2016-06-13
• Study First Posted: 2016-06-16
• Study Start: 2017-02-02
• Primary Completion: 2018-10-22
• Study Completion: 2019-04-17
• Status Verified: 2019-07
• Last Update Submitted: 2019-07-29
• Last Update Posted: 2019-07-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 22

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Sham-RMT	Sham-RMT therapy using modified RMT device	Subjects in control arm will be given an inspiratory and expiratory sham-device and will complete Duke-based and home-based sham-RMT therapy.
Respiratory muscle training (RMT)	RMT therapy using modified RMT device	Subjects in the experimental arm will be given an inspiratory and expiratory RMT device to use during Duke-based and home-based RMT therapy.

Primary Outcome Measures

Name	Time	Method
Change in respiratory strength	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	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.

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States