NCT02801539

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

Completed NA Last updated 30 July 2019

What this trial tests

NA trial testing RMT therapy using modified RMT device in Glycogen Storage Disease Type II in 22 participants. Completed in 17 April 2019.

Timeline

2 February 2017

Primary endpoint
22 October 2018

17 April 2019

Quick facts

Lead sponsor	Duke University
Phase	NA
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	supportive care
Enrollment	22
Start date	2 February 2017
Primary completion	22 October 2018
Estimated completion	17 April 2019
Sites	1 location across United States

Drugs / interventions tested

• RMT therapy using modified RMT device
• Sham-RMT therapy using modified RMT device

Conditions studied

• Glycogen Storage Disease Type II — all drugs for Glycogen Storage Disease Type II →

Sponsor

Duke University

Who can join

18 and older, any sex, with Glycogen Storage Disease Type II. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Respiratory muscle training in late-onset Pompe disease: Results of a sham-controlled clinical trial.
   Jones HN, Kuchibhatla M, Crisp KD, Hobson-Webb LD, et al · · 2020 · cited 14× · PMID 33127291 · DOI 10.1016/j.nmd.2020.09.023
2. Respiratory muscle training (RMT) in late-onset Pompe disease (LOPD): A protocol for a sham-controlled clinical trial.
   Jones HN, Kuchibhatla M, Crisp KD, Hobson Webb LD, et al · · 2019 · cited 5× · PMID 31303277 · DOI 10.1016/j.ymgme.2019.05.001