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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

22 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-02-02

Study Completion Date

2019-04-17

Brief Summary

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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.

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Detailed Description

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Conditions

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Glycogen Storage Disease Type II

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

SUPPORTIVE_CARE

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Respiratory muscle training (RMT)

Subjects in the experimental arm will be given an inspiratory and expiratory RMT device to use during Duke-based and home-based RMT therapy.

Group Type EXPERIMENTAL

RMT therapy using modified RMT device

Intervention Type DEVICE

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

Sham-RMT

Subjects in control arm will be given an inspiratory and expiratory sham-device and will complete Duke-based and home-based sham-RMT therapy.

Group Type SHAM_COMPARATOR

Sham-RMT therapy using modified RMT device

Intervention Type DEVICE

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

Interventions

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RMT therapy using modified RMT device

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

Intervention Type DEVICE

Sham-RMT therapy using modified RMT device

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

Intervention Type DEVICE

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No