Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

Interested in this Trial?

Contact Us

Trial Status Completed

Trial Identifier

NCT02354664

Condition

Pompe Disease

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

This study intends to evaluate dynamic respiratory motor performance as a valuable measure of pulmonary function in adults with late-onset Pompe disease. The investigators will adopt a strategy that includes comprehensive evaluations of respiratory volume, flow, and timing parameters during resting and loaded breathing. These evaluations will then be associated to the standard clinical measure of maximal inspiratory pressure, the static inspiratory muscle function, as well as magnetic resonance imaging of thoracic expansion and diaphragmatic descent at rest and with exertion. Outcomes in participants with late-onset Pompe disease will be contrasted to the function of age- and gender-matched control subjects. This approach will enable the investigators to evaluate the relationship between dynamic diaphragmatic function and respiratory motor function.

Eligibility Criteria

Birth Sex

All

Age

20 Years to 65 Years

Healthy Volunteers

Yes

Drug/Treatment:

Thoracic MRI, Spirometry, Inspiratory Load Compensation, Maximal Inspiratory Pressure, Resting Breathing Pattern, Respiratory Muscle Endurance Test

Study Type:

Observational

Number of Participants:

14

Study Started:

2015-04

Study Updated:

2024-06-24

Trial Locations

  • University of Florida

    Gainesville, Florida, United States

Inclusion Criteria

  • Confirmed diagnosis of Pompe disease OR
  • Be an age and gender-matched unaffected control subject

Exclusion Criteria

  • Pre-existing obstructive lung disease or asthma
  • Forced vital capacity (FVC) 80% of age/gender predicted values
  • Inability to travel to the study site
  • Requirement for positive pressure ventilator support when awake and upright
  • Participation in longitudinal studies that may independently alter lung function
  • Presence of any other chronic medical condition that, in the opinion of the investigator, would make the subject unsuitable for the stud

Contact BioMarin Clinical Trials

Please complete and submit the following form to express interest in participating and request more information about one of our clinical trials.

By completing this form and clicking the send button, you understand and hereby consent to storage of your personal information, including within the United States and Europe, which will be accessible by BioMarin for purposes such as responding to your request, quality control, fulfilling compliance obligations, and assisting with products, services, or clinical trials. Depending on where you live, you may have the right to request access to, removal, or correction of your personal information held by BioMarin. You may submit your request via the contact information and/or webform located within the full BioMarin Privacy Policy.

*required fields

"*" indicates required fields

This field is hidden when viewing the form
This field is hidden when viewing the form