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

Glycogen Storage Disease Type II Clinical Trial

Official title:

Respiratory Muscle Training in Late-Onset Pompe Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02801539
• Other study ID #: Pro00068728
• Secondary ID: 1R21AR069880-01
• Status: Completed
• Phase: N/A
• Start date: February 2, 2017
• Est. completion date: April 17, 2019

Study information

• Verified date: July 2019
• Source: Duke University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: April 17, 2019
• Est. primary completion date: October 22, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Glycogen Storage Disease
• Glycogen Storage Disease Type II

Intervention

Device:
• RMT therapy using modified RMT device
The exercises provided by the RMT device is intended to strengthen breathing muscles.
• Sham-RMT therapy using modified RMT device
The exercises provided by the Sham-RMT modified device is not intended to strengthen breathing muscles.

Locations

Country	Name	City	State
United States	Duke University Medical Center	Durham	North Carolina

Sponsors (2)

Lead Sponsor	Collaborator
Duke University	National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in respiratory strength	RMT to sham-RMT comparison of maximum inspiratory pressures (MIP). Change pretest to posttest is primary measure (baseline to 12 weeks).	baseline, 12 weeks, 6 months, 9 months
Primary	Feasibility of sham-RMT, as measured by program adherence	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.	12 weeks
Secondary	Change in maximum expiratory pressures (MEP)	Change pretest to posttest	baseline, 12 weeks, 6 months, 9 months
Secondary	Change in 6-minute walk test (6MWT)	Change pretest to posttest	baseline, 12 weeks, 6 months, 9 months
Secondary	Change in Gait, Stairs, Gower, and Chair (GSGC) scale	Change pretest to posttest	baseline, 12 weeks, 6 months, 9 months
Secondary	Change in peak cough flow (PCF)	Change pretest to posttest	baseline, 12 weeks, 6 months, 9 months
Secondary	Change in Rasch-built Pompe-specific Activity Score (R-PAct)	Change pretest to posttest	baseline, 12 weeks, 6 months, 9 months
Secondary	Change in diaphragm thickness	change pretest to posttest	baseline, 12 weeks, 6 months, 9 months