A new clinical trial assessing the safety and feasibility of high-dose inspiratory muscle training delivered remotely in patients with late-onset Pompe disease has been launched at Duke University in Durham, North Carolina. The study will also determine the effects of high-dose inspiratory muscle training on respiratory and patient-reported outcomes.

The trial aims to recruit 29 patients, aged 18 years and above, with a confirmed diagnosis of late-onset Pompe disease. Inspiratory muscle training will be performed 3 times a week for 26 weeks using a mobile device called Pr02, which measures and increases respiratory strength and performance through resisted breathing exercises.

“Our hypothesis is that high-dose [inspiratory muscle training] is necessary to produce meaningful changes in respiratory muscle strength and other outcomes in participants with [late-onset Pompe disease],” the investigators wrote. 


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The primary outcome measure of the trial is the change in maximum inspiratory pressure from baseline at weeks 15 and 30. Secondary outcome measures include changes in measures of respiratory and motor functions, fatigue, sleep, communication, and swallowing.

The estimated completion date of the trial is August 2024. 

Pompe disease is divided into 2 main categories based on the age of onset and whether cardiomyopathy is present. Infantile-onset Pompe disease is the most severe form and affects children under the age of 12 months. Late-onset Pompe disease, on the other hand, is less severe and symptoms usually appear after 1 year of age. Cardiac involvement is uncommon. Symptoms include scoliosis, lumbar hyperlordosis, loss of mobility, and breathing difficulties caused by muscle weakness.

Read more about the symptoms of Pompe disease

It is of great importance to identify ways of addressing respiratory function that are easy to implement and cost effective to reduce disease burden and improve mobility as well as health-related quality of life.

Reference

High dose inspiratory muscle training in LOPD. Clinicaltrials.gov. June 24, 2022. Accessed July 6, 2022.