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

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Duke University

ClinicalTrials.gov Identifier:

NCT01701154

First received: October 3, 2012

Last updated: January 16, 2013

Last verified: October 2012

History of Changes

Purpose

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.

Condition	Intervention
Pompe Disease	Other: respiratory muscle strength training

Study Type:	Observational
Study Design:	Observational Model: Cohort Time Perspective: Prospective
Official Title:	The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease

Resource links provided by NLM:

Genetics Home Reference related topics: glycogen storage disease type IX Pompe disease Schindler disease succinic semialdehyde dehydrogenase deficiency

U.S. FDA Resources

Further study details as provided by Duke University:

Enrollment:	11
Study Start Date:	December 2010
Study Completion Date:	December 2012
Primary Completion Date:	December 2012 (Final data collection date for primary outcome measure)

Groups/Cohorts	Assigned Interventions
Pompe Adults and children with Pompe disease.	Other: respiratory muscle strength training

Detailed 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.

Eligibility

Ages Eligible for Study:	3 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Adults and children with Pompe disease

Criteria

Inclusion Criteria:

include skin fibroblast acid α-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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01701154

Locations

United States, North Carolina
Duke University Health System
Durham, North Carolina, United States, 27710

Sponsors and Collaborators

Duke University

Investigators

Principal Investigator:

Harrison Jones, PhD

Duke University

More Information

No publications provided

Responsible Party:	Duke University
ClinicalTrials.gov Identifier:	NCT01701154 History of Changes
Other Study ID Numbers:	Pro00024468
Study First Received:	October 3, 2012
Last Updated:	January 16, 2013
Health Authority:	United States: Institutional Review Board

Keywords provided by Duke University:

Pompe respiratory

Additional relevant MeSH terms:

Respiratory Aspiration
Glycogen Storage Disease Type II
Respiration Disorders
Respiratory Tract Diseases
Signs and Symptoms, Respiratory
Signs and Symptoms
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic

Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on May 16, 2013

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