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

Duke University22 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

SUPPORTIVE_CARE

Masking

DOUBLE

Enrollment

Conditions

Glycogen Storage Disease Type II

Interventions

RMT therapy using modified RMT deviceDEVICE

The exercises provided by the RMT device is intended to strengthen breathing muscles.

Sham-RMT therapy using modified RMT deviceDEVICE

The exercises provided by the Sham-RMT modified device is not intended to strengthen breathing muscles.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (1)

Duke University Medical Center

Durham, North Carolina, United States

Outcomes

Primary Outcomes

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).

Time frame: baseline, 12 weeks, 6 months, 9 months

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.

Time frame: 12 weeks

Secondary Outcomes

Change in maximum expiratory pressures (MEP)

Change pretest to posttest

Time frame: baseline, 12 weeks, 6 months, 9 months

Change in 6-minute walk test (6MWT)

Change pretest to posttest

Time frame: baseline, 12 weeks, 6 months, 9 months

Change in Gait, Stairs, Gower, and Chair (GSGC) scale

Change pretest to posttest

Time frame: baseline, 12 weeks, 6 months, 9 months

Change in peak cough flow (PCF)

Change pretest to posttest

Time frame: baseline, 12 weeks, 6 months, 9 months

Change in Rasch-built Pompe-specific Activity Score (R-PAct)

Change pretest to posttest

Time frame: baseline, 12 weeks, 6 months, 9 months

Change in diaphragm thickness

Data from ClinicalTrials.gov

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