The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease

Pompe Disease Clinical Trial

Official title:

The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01701154
• Other study ID #: Pro00024468
• Status: Completed
• Start date: December 2010
• Est. completion date: December 15, 2012

Study information

• Verified date: May 2023
• Source: Duke University
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The purpose of the study is to determine if respiratory muscle strength training will be beneficial for inspiratory and expiratory muscle strength in adults and children with Pompe disease.

Description:

Respiratory muscle weakness results in substantial morbidity and mortality in individuals with almost all forms of neuromuscular disease (NMD), including both the infantile and adult phenotypes of Pompe disease. Although individual patterns of involvement vary, respiratory weakness in Pompe disease typically affects both the inspiratory and expiratory muscle systems. Our pilot data in two individuals with late-onset Pompe disease suggest that RMST may be a treatment for the progressive respiratory weakness encountered in this condition.

Purpose of the Study

• Determine the effect of respiratory muscle strength training on maximum inspiratory pressure, maximum expiratory pressure, upright % predicted forced vital capacity, and aspects of cough function.

• Determine the effect of RMST on functional outcome measures of gross motor function appropriate for individual participants in terms of age and motor skills.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. completion date: December 15, 2012
• Est. primary completion date: December 15, 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 99 Years

Eligibility

Inclusion Criteria:

• include skin fibroblast acid a-glucosidase (GAA) activity =1% of the normal mean, a minimum age of 3 years, the ability to participate in an intensive RMST research program, and the ability to maintain a consistent amount of nonresearch related physical activity over the course of the study.

Exclusion Criteria:

• include medical problems which preclude meaningful participation in the study, the inability to perform high effort respiratory tasks using maximum intensity, respiratory weakness so profound that RMST cannot be completed at the minimum pressure thresholds of available respiratory trainers, and the inability to safely perform the protocol. For example, high effort respiratory tasks are occasionally associated with mild, transient dizziness which quickly dissipates following a brief rest. If severe and/or prolonged dizziness were to occur in a particular case, then exclusion from the study would be necessary.

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Pompe Disease

Intervention

Other:
• respiratory muscle strength training

Locations

Country	Name	City	State
United States	Duke University Health System	Durham	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Duke University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in maximum inspiratory pressure	change from pre-test to post-test	baseline, week 12